EP 21: May TL;DR and Dazzle4Rare 2023 Housekeeping

Show Notes

We're kicking off with a bang! May is jam-packed with event, awareness days, and news! Without further ado, let’s do the darn thing and get to Rare and Relevant, Your TL;DR!

First up, it's Huntington's Disease Awareness Month, Acute Disseminated Encephalomyelitis (ADEM) Awareness Month, ALS Awareness Month, Cystic Fibrosis Awareness Month, Ehlers-Danlos Syndrome and Hypermobility Syndrome Awareness Month, Ichthyosis Awareness Month, Myositis Awareness Month, Prader-Willi Syndrome Awareness Month, Progressive Supranuclear Palsy (PSP) Awareness Month, and PTEN Hamartoma Tumor Syndrome Awareness Month, all happening from May 1st to 30th.

Speaking of PTEN awareness month, back in November of 2022 I spoke with Cowden Syndrome / PTEN syndrome patient advocate and men’s mental health advocate, David Ross where he shared a little about the association between these conditions.

If you’d like to hear the rest of the episode after this one, the link will be in the show notes.

[https://signalise.podbean.com/e/ep8-david-ross/]

Stil in May, the 15th marks International Mucopolysaccharidosis Awareness Day and Tuberous Sclerosis Complex (TSC) Global Awareness Day. And don't forget CDG Awareness Day on May 16th, dedicated to raising awareness for a family of rare metabolic diseases that are often overlooked by the medical and scientific communities.

If that’s not enough awareness for you in May, we also have World Neurofibromatosis Awareness Day on May 17th, dedicated to raising awareness for this condition, causing tumors to grow on nerves throughout the body.

On May 19th, we have #Red4Veds, a social media campaign where people are encouraged to wear red and share photos on social media to raise awareness for Vascular Ehlers-Danlos Syndrome, a rare genetic disorder that affects the connective tissues in the body. vEDS carries with it a shortened life-span with most folks rarely surpassing their 40th birthday. If you have a family history of aortic dissection, tears or eruptions of other organs, please see your GP.

And for those in Wisconsin, USA mark your calendars for Rare Disease State Advocacy Day on May 17th from 3:30pm to midnight, organized by the Every Day Life Foundation. This is a day for Wisconsin residents impacted by rare diseases to come together, meet with state legislators, and share their stories to help advance the policy priorities of the rare disease community.

What about PS? Podcast

Did you know that friend of the podcast and first guest, Sam Fillingham, has started a Poland Syndrome podcast alongside host Giselle Barbosa?

The podcast is called “What about PS?” and will be hosted primarily by Giselle. She is a PIP-UK ambassador, an entrepreneur, and an athlete!

According to the press release, “ (The) podcast aims to shine a light on Poland Syndrome, a rare disease, and the lived experience of the Poland Syndrome community. Each episode, Giselle will explore inclusive and varied experiences of people living with Poland Syndrome with a variety of guests, from Paralympian Kim Daybell, Former Cricketer Lewis Hatchett, Australian Gymnast Clay Mason Stephens to medical professionals, bra companies and the most amazing members of our community. With engaging interviews, thought-provoking discussions, and the inclusive experience of the Poland Syndrome Community. The What about PS? podcast promises to be a must-listen for anyone curious about Poland Syndrome. Including the Poland Syndrome community, their friends and families, medical professionals and the rare disease community.”

[Insert Whatsapp clip from Sam if she sends one]

Whether you are affected personally by Poland Syndrome or not, I have no doubt the podcast will be interesting and engaging! You can find them wherever you get your podcasts, just search “What about PS?”

Very quickly, also in Poland Syndrome news, a little boy here in the UK named Charlie hosted a fun-run for Poland Syndrome. He’s a big fan of PIP-UK and the amazing things they do for kids like him. He raised over £844, roughly 1,560 US Dollars or 87392 Indian Rupies, for PIP UK. He reached 168% percent of his goal! Well done, Charlie and to the 32 folks who donated to his fun run for Poland Syndrome Support UK. A link will be in the show notes.

[https://www.justgiving.com/page/pip-uk-poland-syndrome-charity-charliesrun]

News Segment

All links will be in the show notes so make sure to subscribe on your podcasting app of choice or subscribe to our YouTube Podcast. You can find us by searching Dazzle4Rare on any of these platforms.

Sanford Research hosts the 13th Annual CoRDS Great Plains Rare Disease Summit

From the 24th April press release, “Sanford Research will host the Sanford CoRDS 13th Annual Great Plains Rare Disease Summit May 11-12 in Sioux Falls. This year’s event theme is, ‘Rare Neurodevelopmental Disorders.’”

The annual summit on rare diseases aims to bring together researchers and those affected by rare diseases to collaborate and educate people on advancements in the field of pediatric neurodevelopmental disorders. The event kicks off with opening remarks from Ashish Gupta, followed by presentations from rare disease advocates Amber Freed and Allison Bones. Thursday's sessions are open to patients, families, and those in the community impacted by rare diseases.

[source: https://news.sanfordhealth.org/news-release/sanford-research-to-host-great-plains-rare-disease-summit/]

Shout-out to Allison if you’re listening!

On to topic of CoRDS and Sanford Research

Sanford Health is a non-profit health care provider in the US. They’ve created a Rare Disease Registry to gather data on rare conditions. The registry aims to improve the understanding of rare diseases and accelerate research and treatment development. The registry is open to anyone in the US with a rare condition and is free to participate in. Participants can opt to share their medical history, symptoms, and genetic information to help researchers get a better understanding of their condition. The registry also includes a biobank where participants can donate samples for further research. So far, the registry has enrolled over 2,000 participants and is actively seeking more.

What’s a biobank, you may be asking?

A biobank in rare disease is a repository that stores biological samples (such as blood, tissue, urine, or saliva) and associated data from individuals with rare diseases. The purpose of a biobank is to provide a resource for researchers to study the underlying causes of rare diseases, develop new diagnostic tools and treatments, and ultimately improve patient outcomes.

Biobanks typically collect samples from individuals with rare diseases and their family members, as well as from healthy individuals for comparison. The samples are carefully stored and labeled, and the associated data (such as medical histories, genetic information, and demographic information) are recorded in a database.

Biobanks are critical for advancing rare disease research because they provide researchers with access to a large number of rare disease samples, which would be difficult to obtain on an individual basis. They also help to standardize sample collection and data recording, which makes it easier to compare data from different studies and identify commonalities and differences between rare diseases.

We’ll drop a link in the show notes for more info.

[source: https://research.sanfordhealth.org/rare-disease-registry]

New study discovers over 800 genes causing 60 rare genetic disorders in over 5,500 individuals, highlighting need for increased research participation for underrepresented groups

The study called Deciphering Developmental Disorders looked at 5,500 people in the UK and Ireland. They found that over 800 genes were involved in causing about 60 conditions, with three-quarters caused by spontaneous mutations. This research can help doctors make faster and more accurate diagnoses for people with these conditions.

The study also found that families of non-European ancestry had lower chances of getting a correct diagnosis, spotlighting the need for increased research participation from under-represented groups. By encouraging participation from people of diverse ethnic and economic backgrounds in research studies, scientists can gain a better understanding of how genetics and ethnicity are related, which will lead to better diagnostics and treatment options. This underscores the importance of events like EDIRA or Equality and Diversity in Research Association. Check out EP15 for more info on EDIRA.

[source one: https://www.ddduk.org/updates.html]

[source two: https://www.ndph.ox.ac.uk/news/5-550-people-diagnosed-with-rare-genetic-diseases-in-major-research-study]

AstraZeneca drops $855 million dollar drug

AstraZeneca, a drug company, has abandoned a rare disease drug candidate worth $855 million after two phase 2 trials failed. The drug was originally successful in a larger phase 3 trial, but the company found out that it didn't work as well as they thought. The drug was meant to eliminate copper from the body in patients with Wilson disease, but the phase 2 trials didn't show that it did so. The company decided it would take too long to develop the drug further and decided to stop investing in it.

Conferences

All-around likable guy, Daniel DeFabio will be at BIO Boston screening five of YOUR rare disease films at the conference. Follow Daniel on Instagram at https://www.instagram.com/disorderrarediseasefilms/.

Incidentally, BIO Boston is also on the Dazzle4Rare Community Calendar. If you’re not already subscribed to the calendar and adding YOUR community events of interest, what are you waiting for?

Visit dazzle4rare.net for the online version of the events and awareness calendars. To subscribe, email me for details.

Solve-RD

The H2020 funded flagship EU project, Solve-RD, is making strides towards solving rare disease mysteries where a molecular cause is not yet known. They’ve achieved a successful Seeding Grant project where they’ve used zebrafish to confirm AMFR as a cause for Hereditary Spastic Paraplegia. You can find this on their website.

The groundbreaking project brings all of us one step closer to finding the answers needed in rare diseases. Learn more about Solve-RD and their ongoing efforts to solve rare diseases at solve-rd.eu.

Can Gene Therapy Help Children Walk?

Duchenne muscular dystrophy is a genetic disorder that leads to muscle degeneration, resulting in young patients like Conor in this story requiring full-time wheelchair use by the age of 10 years of age.

However, with a gene therapy from Pfizer due for approval from the Food and Drug Administration next month, the future is looking brighter for children with the disease. The first patient, Conner Curran, underwent the trial treatment five years ago, which triggers muscle cells to produce the dystrophin protein that is essential for muscle function, and has since been able to retain mobility. The gene therapy does not provide a cure, but it does provide a "meaningful benefit" for more than five years, giving the boys the opportunity for a better life.

I say, “boys” here beacause according to “DMD generally affects boys because the dystrophin gene is on the X chromosome. Chromosomes are the parts of your cells that contain your genes. Boys only have one X chromosome.”

[https://www.cedars-sinai.org/health-library/diseases-and-conditions---pediatrics/d/duchenne-muscular-dystrophy.html#:~:text=DMD generally affects boys because,only have one X chromosome.]

Duchenne's affects 12,000 to 15,000 children in the US and 300,000 worldwide. It is usually genetic, passed down from a mother to a son, as the gene sits on the X chromosome, which determines sex. The disease is caused by a mutation in the dystrophin gene, which leads to the inability to produce the essential protein needed for muscle function. Without dystrophin, muscles cannot repair themselves, leading to the accumulation of damage over time.

Four companies are currently developing gene therapies for Duchenne's, and Sarepta Therapeutic's drug SRP-9001 will be the first to be considered by the FDA on May 12th. Although gene therapy is exciting, long-term prognosis for patients remains uncertain as each disease requires a distinctive gene therapy and each company has a slightly different approach. While there is still more work to do, significant improvements have been made in the last eight years, with 50 treatments in the pipeline, promising hope for the future for those affected by Duchenne's.

----

Don't miss an episode of Signalise! Be sure to ✔️FOLLOW or ✔️SUBSCRIBE via your podcast app of choice. You can also follow #Signalise news on @Dazzle4Rare on Facebook , Instagram , and Twitter. You can find host Kimberly on LinkedIn at @kattague.

What is Signalise: a Dazzle4Rare Podcast?

The Dazzle4Rare event and Signalise podcast amplify the voices of rare disease and associated communities by sharing their stories, new, events, and more. Working together, we have strength in numbers, amplifying our critical messages.

We feature guests and discuss relevant topics for rare disease patients, caregivers, and those in the URCIID community.

Hi I'm your host, Kimberly
Thomas-Tague and

you're listening to
Signalise: a Dazzle4Rare

podcast. Whether you're a patient advocate,
caregiver, or clinician, Signalise as your

source for good news, personal stories,
events, and the things that rare and associated

communities care about,
follow Signalise and

Dazzle4Rare at D A Z Z
L E, the number FOUR,

R A R E

on Facebook, Instagram and Twitter, where we'll post episode links, updates, and.

More we welcome to episode
21. Whether this is your

first or your 21st episode.
Thanks for taking the time.

That means a lot to me and
the folks whose news events

and guest appearances
are featured in the podcast.

If you'd like to share something
with us cheque, the show

notes for details on how to make
contact now on with your TLDR.

Rare and relevant.

First up, we have Huntington's disease.

Acute disseminated encephalomyelitis,
or ADEM ALS awareness, cystic

fibrosis, ellers, danlos and
hypermobility syndromes, respectively,

each theosis awareness month,
myositis Prater, Willy syndrome,

progressive supranuclear policy or
PSP and P-10 hamartoma tumour.

Syndrome Awareness Month, also
associated with cowden's disease.

It's all happening from the
1st to the 30th of this month.

Speaking of P-10 Awareness
month, back in November of 2022,

I spoke with Cowden syndrome,
patient advocate and men's

mental health advocate
David Ross, where he shared a

little bit about the association
between these conditions.

PTEN Hamartoma Tumour syndrome
is [...] Cowden Syndrome comes under

the umbrella of. People carries
an increased risk of getting certain

cancers, autism and developmental
delays. Also, some of the other

things that are associated with
my rare disease whilst you're given.

The diagnosis of Cowden syndrome. It does.

Fits under this umbrella.

It sent home at home Tumour
syndrome, the reason why

it's called PTEN is because
of the P-10 gene, which

is linked to the cell
overgrowth, which cause the for

example, the increased
risk of getting some cancers.

If you'd like to hear more from David in that episode, the link will be in the show.

Still, in May we have the 15th, which
marks international mucopolysaccharidosis.

I hope I said that right Awareness
Day and the tuberous sclerosis complex

known as TSC and the Global Awareness
Day is also on the 15th. Don't forget,

CDG Awareness Day on the 16th
and it is dedicated to raising awareness.

For a family of rare
metabolic diseases that are

often overlooked by medical
and scientific community.

If that's not enough, in May we
also have world Neurofibromatosis

Awareness Day on the 17th. This
state is dedicated to raising awareness

for this condition, which causes
tumours to grow on the nerves throughout

the body. On the 19th of May we
have hashtag red RED the number 4.

V EDS. Red 4.

This is a social media campaign where
people are encouraged to wear red and share

photos on social media to raise awareness
for vascular Elers Danlos syndrome V

DS carries with it a short lifespan,
which most folks really surpass their 40th

birthdays. If you have a family history
of aortic dissection, tears or eruptions.

Of the organs, please see your GP.

Now to bound out awareness days, we have
one in Wisconsin, USA, so market calendars

for rare disease state advocacy day on
the 17th of May from 3:30 PM to midnight.

This event is organised by Everyday Life
Foundation. This is for Wisconsin residents

who are impacted by rare diseases to
come together, meet with state legislators.

And share their stories
to help advance policy

priorities for the rare
disease community.

And Speaking of calendars, if you
didn't know, we have two dazzle for our

calendars. Both are public and are accessible
to you anytime, anywhere on the go.

The first calendar we have
is our community events

calendar, which is
maintained by myself and our

dazzle for rare network.
If you would like to add

your webinar, your podcast
or your other reoccurring.

Event or one time events.
We also have an awareness

days calendar and it also
has a couple of other days for

fun thrown in there and
you can add to that calendar

as well if your awareness
date is not already there.

For more details you can look
in the show notes or reach out

to me and now for something
completely different. What about P?

Yes. Well, what about PS
is a podcast. Did you know

that friend of this
podcast, signalised and our

first signalised guest,
Sam Fillingham has started

a Pollen syndrome podcast
with host Giselle Barbosa?

Some filling room. I'm CEO of Pippu carry the pollen syndrome support and network.

Territory they were.

A charity that supports
families affected by a

rare lung difference of
congenital anomaly called

Poland syndrome. The
main symptoms are a missing

and underdeveloped
chest muscles, chest veins.

General underdevelopment in
the chest area and also under

development in the hands and
fingers and the main symptoms.

If you didn't know,
then you know now the

podcast is again called
what about PS and will

be hosted primarily by Giselle. She's a pip
UK ambassador and entrepreneur and an at.

According to the press release, the
podcast aims to shine a light on pollen

syndrome, a rare disease, and the lived
experience of the pollen syndrome community.

Each episode Giselle will
explore inclusive and varied

experiences of people living
with Poland syndrome with a

variety of guests from Para
Olympian Kim Daybell, former

cricketer Lewis Hatchett,
Australian gymnast Clay Mason.

Stephens to medical
professionals, bra companies and

the most amazing members
of our community with engaging

interviews, thought
provoking discussions and the

inclusive experience of the
Poland syndrome community.

The what about PS podcast
promises to be a must listen

for anyone curious about
pollen syndrome, including

the pollen syndrome
community, their friends, family

medical professionals, and
the rare disease community.

One last thing quickly.
Here in Poland Syndrome

News, a little boy here
in the UK named Charlie

hosted a fun run for
Poland syndrome. He's a big

fan of Pipe UK and the
amazing things that they do.

For kids like him, he
raised over 844 pounds,

which is roughly 1560
U.S. dollars, or 87,000.

392 Indian rupees for
Poland Syndrome support UK

he reached 168% of his
goal. Well done Charlie and.

Also well done to the 32 folks who
donated to his fund run for Poland Syndrome

support UK. We'll leave a link in the
show notes and now Forbidden News.

All links will be in the
show notes, so make sure to

subscribe to podcast on your
podcasting app of Choice or

subscribe to our YouTube
podcast. You can find us by searching

a dazzle for rare on any
of these platforms. Sanford.

Research hosts the 13th annual Chords, Great Plains Rare Disease summit.

From the 24th of April press release, Sanford
Research will host the Sanford Chords,

thirteen annual Great Plains rare Disease
Summit, May 11th to 12th in Sioux Falls.

This year's event theme
is rare neurodevelopmental

disorders. The annual
summit on rare diseases aims

to bring together researchers
and those affected by

rare diseases, to collaborate
and educate people.

And advancements in the field of paediatric
neurodevelopmental disorders. The

event kicks off with opening remarks by
Ashish Gupta, followed by presentations

from rare disease advocates Amber Freed
and Alison Bones. Thursday, Sessions are

open to families, patients, and those in
the community impacted by rare disease.

And Allison, if you're listening, shout out
to you on the topic of chords and Sanford

research. Sanford Health is a nonprofit
healthcare provider in the United States.

They have created a rare
disease registry to gather data on

rare conditions. The registry
aims to improve the understanding

of their diseases and
accelerate research and treatment

development. The registry is
open to anyone in the United States.

The rare condition and
it is free to participate.

Participants can opt to
share their medical history,

symptoms and genetic
information to help researchers

get a better understanding
of their condition.

The registry also includes a bio bank where
participants can donate samples for further

research. So far, the
registry has enrolled

over 2000 participants
as actively seeking more.

This is pretty exciting stuff, especially
the biobanking part. If you're not sure and

you're asking yourself what is a biobank,
let me just give you a quick explanation.

A biobank in rare diseases is a
repository that stores biological

samples such as blood, tissue,
urine or saliva, and associated

data from individuals with rare
diseases such as their medical

records or a copy of their
diagnosis or other relevant data.

The purpose of a biobank
is to provide a resource for

researchers to study the
underlying causes of their diseases.

Develop new diagnostic
tools and treatments and

ultimately improve
patient outcomes. Biobanks

typically collect
samples from individuals

with rare diseases and
their families as well.

As well as healthy
individuals for comparison, the

samples are carefully
stored and labelled, and the

associated data such as
medical histories, genetic

information and demographics
are recorded in a database.

Biobanks are critical for
advancing rare disease

research because
they provide researchers

with access to a large
number of rare disease

samples, which would
be difficult to obtain.

On an individual basis, they
also help us to standardise sample

collection and data recording
which makes it easier to compare

data from different studies
and identify commonalities and

differences between rare
diseases. We'll drop a link in the.

Notes a new study
discovers over 800 genes

causing 60 rare genetic
disorders, and over 5500

individuals, highlighting
the need for increased

research participation for
underrepresented group.

The study is called deciphering
developmental disorders,

and it looked at 5500
people in the UK and Ireland.

They found that over 800 genes
were involved in causing about 60

conditions, with three quarters
caused by spontaneous mutation.

This research can be
helpful to doctors to make

faster and more accurate
diagnosis for people

with these conditions.
The study also found

that families of non
European ancestry had lower

chances of getting a
correct diagnosis, spotlighting

the need for increased
research, participation

from underrepresented
groups by encouraging

participation from people
of diverse ethnicities.

And economic backgrounds in
research, scientists can gain a better

understanding of how these factors
are related or may interplay, and this will

hopefully lead to better diagnostic
and treatment options. This underscores

the importance of events like
adira, the equality and diversity and.

Search association cheque out episode 15 For
more information on Adira. This next piece

of news is kind of bonkers, but I'm expecting
there's probably a good reason for this.

AstraZeneca drops and $855 million
drug AstraZeneca, a drug company,

obviously has abandoned a rare
disease drug candidate worth 855.

Million U.S. dollars after phase
two trials failed. The drug was

originally successful in a larger
phase three trial, but the company

found out that it didn't work
as well as they had thought. The

drug was meant to eliminate
copper from the body in patients with.

'S disease. But the
phase two trials didn't

show that it did that
the company decided it

would take too long to
develop the drug further

and has decided to
stop investing in it.

Upcoming conferences. Yay. I know a
lot of folks listening, probably do attend

conferences, whether they attend them
in person or online. If this isn't you.

Don't worry, it's a quick segment.

All round Michael Guy Daniel
Defabio will be at the bio Boston

Conference screening five of your
rare disease films at the conference.

Follow Daniel on Instagram
at instagram.com/disorder rare

disease films. Incidentally,
Bio Boston is also on the

dazzle of Rare community
calendar that we mentioned

earlier. If you're not already
subscribed to the calendar.

Please do so now get in touch
adding your community events of

interest can only benefit you.
So what are you waiting for?

Visit dazzlefora.net, which at the time
of recording is down for maintenance

but should be up soon if not reach
out to me. Info is still in the show note.

Next up we have kale gene therapy
help children walk. So if you are not

familiar with the condition Duchenne
muscular dystrophy or DMD, it's a

genetic disorder that leads to
muscle degeneration resulting in young

patients like the child in this story
needing to use a wheelchair full time.

Sorry about the age.

Ten. However, in this case with gene therapy
from Pfizer, which is hopefully due to

be approved either this month or next,
the FDA or Food and Drug Administration is.

To be releasing this drug and the
future looks bright for children like

this little boy. The first patient Connor
Kieran, underwent the treatment.

Five years ago.

This treatment triggers cells to produce
dystrophin protein, which is essential

to muscle function and has since been
able to help Connor retain his mobility.

The gene therapy doesn't provide a
cure, but it does provide a meaningful

benefit for up to five years, giving
boys the opportunity for a better life.

Now I say boys because
according to Cedar

Sinai, DMD generally
affects boys because of the

dystrophin gene which
is on the X chromosome.

Duchenne affects 12,000
to 15,000 children in the US.

And 300,000 worldwide,
it's usually genetic in

nature, passing down
from a mother to a son.

As the gene sits on the
X chromosome, as we've

just said, the disease
is caused by a mutation

in the dystrophin
gene, which leads to the

inability to produce
essential proteins needed

for muscle function without the dystrophin
muscles cannot repair themselves leading to.

The accumulation of damage overtime
at time of recording four companies are

currently developing gene therapies
for decisions. The company's Sarepta

Therapeutics has a drug called SRP-9001,
and it will be the first to be considered

by the FDA on the 12th of May. So in
just a few days, although gene therapy.

Is exciting long term prognosis remains unknown.

Certain each disease requires A distinctive
gene therapy, and each company has their

own approach and their own timelines where
there is still more work to do, significant

improvements have been
made, with 50 treatments

currently in the drug
development pipeline,

and that makes for a promising future,
hopefully for children affected by Duchenne.

Last news item we have is solve
Rd, the H2020 funded flagship EU

project. Solve Rd is making strides
toward solving rare disease mysteries

where a molecular cause is not yet
known. They've achieved a successful

seed funding grant project where
they've used zebra fish to confirm a.

MFRAMFR.

As a cause for hereditary
******* paraplegia, you

can find more about this
on their website, the ground

Breaking Project brings
all of us one step closer

to finding the answers
needed in rare diseases.

Learn more about solve
Rd and their ongoing efforts

at their website, which
is solve SOLV E-RD dot.

You and that's all she wrote folks for.

This TLDR, rare and relevant,
and now for a bit of dazzle for rare

housekeeping. The dazzle for our
website has been down for a few weeks.

In all honesty, I was
frustrated at not finding any

WordPress templates that I
liked and decided to go back to

creating something more custom
because WordPress has changed

a lot since I did any custom
alterations or any website.

I'm in the process of getting the D4
R 2023 registration back up on the

page. By the time you hear this episode,
the page may be back up without the

registration function temporarily while
I add the support for the registration

process back into the website. This
is usually fairly easy stuff to do and.

Warning excuses ahead, but
with Easter and the bank holidays

and the teachers strike and
the inset days and the King's

coronation amongst some other
personal things that have happened,

I haven't been able to
restore the functionality just yet.

I do believe the new
version of the site, at

least aesthetically,
will be much nicer. Also,

working on cleaning up the content and trying
to distil what dazzle for rare is about.

On that note.

If you are still unsure what Dazzle Ferrer actual.

Is I've been asking folks to
help me out in terms of explaining

what the impact of reaching
the social networks of others is.

You'll know that content creators often
say share my posts. It really helps me out,

but how does it help people out? So
basically rare and associated organisations?

Team up with one another
to get their message

out to more people. We
do this by exchanging

messages. I usually say
Twitter friendly message

because Twitter has
been our biggest platform.

But who knows where it'll be this year?
By working together, all of us can reach a

larger audience and get more attention
to our specific causes or our communities.

If there is a downside to sharing posts
with each other and in good faith sharing,

you know theirs with your audience and
vice versa, then someone pointed out to me.

We keep the event to one week so
that folks in our community and your

community aren't fatigued by hearing
or seeing messages from others, but it's

a great way to help folks in your
community by educating them on something

they may not know anything about
or that may impact them in some way.

And that, you know, I've
heard from people over the

years that that has exactly
been the case or that they

didn't think that there
was much understanding of

their condition outside of
their Facebook community.

And then when they've seen people sharing
about it on during Dazzle Ferrer, they've

just been gobsmacked, which is great. I
love receiving those types of messages, so.

When you reach people through someones
social network, it's called social media

amplification. I like to call it signal
boosting. That's why this is called signal.

Guys, it's like when you tell a
friend something cool that you saw on

social media and they share it with
their friends and so on and so forth.

Sometimes it picks up steam
and becomes viral, and sometimes

it doesn't. By getting other
organisations and influencers

quote unquote to share your content,
where does these organisations

and associated organisations
can amplify these messages?

And reach a wider audience.
I think that's also why it's

really important to put some
care into crafting your message,

because if you only have a
few words to get across what

you're doing to a complete
stranger, you better make it count.

That's what I say. So this
idea can help build a community

of supporters and advocate
who care about rare disease in

general and who actually want
to share your messages and help

others by allowing you to reach
their audience and vice versa.

You guys have all heard me
say that we need to stop preaching

to the choir. If you're not
familiar with that turn of phrase.

Preaching to the choir,
mean speaking to an audience

that already agrees with
you or is already aware of.

Issue in this case with
rare disease organisations

or associated communities
working together on

social media. The goal
is to reach new audiences

beyond the folks who
are already following them.

So rather than just speaking to the
folks who are already aware of the issue

or the issues in your community,
you're reaching new people who may not.

Be aware by leveraging
the social media networks

of others rare disease and
associated organisations

can reach new people,
build larger communities

of supporters who may
not have been aware of

before, and this can help
raise awareness for rare

diseases, can increase
the number of people who

are actively engaged in
advocating for improved

care, and help support
individuals and families.

It's it's a novel concept
now. There are some

historical examples of
movements or causes that utilise.

Social networks to amplify their message
and build a larger community of supporters.

Because Tom from Myspace did not invent
social network, they existed before Myspace.

So one example is the
civil rights movement. During

the 1960s, civil rights
activists use a variety of

tactics to spread their
message and build support for

their cause, including
organising rallies and protests.

Speaking at public events and
working with media outlets to get

coverage, they also leverage
social media networks in person.

Building grassroots
supports for their cause by

working with local
communities, churches and other.

The civil rights activists were
able to build a network of supporters

who could help spread the message
and mobilise others to take action.

Another example is the AIDS
activism movement during the

80s and 90s, activists in the
movement used a variety of

tactics to raise awareness of
the AIDS epidemic and advocate

for improved care and support
for people living with HIV.

They also utilised social networks in person to connect others who were affected by.

Disease. There were a
lot of ways to share these

stories in these messages.
It happened in magazines. It

happened in leaflets,
people at social events talking

about it and people
inviting each other to events.

You know, it's a powerful
thing to, you know, recruit the

people in your life to help.
So by sharing these stories.

By organising events by
working with media outlets to get

coverage activists for the OR
the HIV AIDS community were able

to build a powerful network who
were passionate about fighting

for better care and support
for people living with HIV.

These examples demonstrate the
power of social networks, not necessarily

social media social networks,
to build grassroots support for.

Laws and amplify the message, and that's
exactly what dazzle Harare wants to do. By

leveraging the collective
reach of folks and

organisations and
causes, we can spread the

message to a wider
audience and build a

community of supporters
who are passionate about

rare disease and
associated conditions. So

that's what we're doing.
I hope that made sense.

And I'm going to leave it to.

You thanks for joining me today.

Thanks for listening to
this week's episode of

Signalise, a Dazzle
for our podcast. To stay

up to date on the
podcast and Dazzle Frere,

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