https://t.co/VRn1mIVJ5V

https://t.co/1KA1vGWwK2

@EveryLifeOrg

@SenatorBennetCO @SenMullin @RepCastor @RepMcCaul @RepFrankPallone @RepGuthrie @RepBuddyCarter @US_FDA @RareAdvocates

Let’s get the Pediatric Priority Review Voucher (PPRV) done — and bring hope to kids with rare diseases like LGMD. 🇺🇸 #PPRV #RareDisease #TeamDion #BipartisanHope #AdvocacyInAction

A 9-year-old dressed as her favorite President with one mission: 👉 Tell Congress & the White House to #GiveKidsAChance

Check cosponsors: H.R.1262: https://t.co/Nd4mlnCg1I S.932: https://t.co/ipVHnvHD83 #GiveKidsAChance #RareDiseaseAwareness #KidsDeserveCures

Grateful to lawmakers backing the Give Kids a Chance Act (S.932 / H.R.1262)! My brother & I have a rare disease — we need more treatments made for kids like us. Let’s #PassTheBill & give kids a real shot at a cure.

[GENE THERAPY] 📢@GenethonFr is delighted with the partnership between its spin-off @AtamyoTX and the Dion Foundation to extend the clinical trial in the United States with ATA-200, a #GeneTherapy for limb-girdle muscular dystrophy type 2C/R5 (LGMD2C/R5). #lgmd#ClinicalTrial

@DionFund is proud to announce this new partnership with @AtamyoTX to expand their first-in-human trial of ATA-200 #genetherapy to treat LGMD2C/R5 in the US. Read full press release here: https://t.co/XKBKVqBIO3 #LGMD #MuscularDystrophy

@HouseCommerce

Thank you @cathymcmorris for sharing our story #energyandcommercecommittee and @RepLoriTrahan for your work on HR 7384 #creatinghopereauthorizationact to help children like Peter & Maggie living with rare pediatric diseases 🙏🏻

Remarkable performance from Buddy! (old photo) #hero #fearless #savant #virtuous #legend #statue for buddy $SRPT

NEWS: An update on the outcome of today’s FDA Advisory Committee Meeting on SRP-9001, our investigational gene therapy for Duchenne muscular dystrophy. Read more here: https://t.co/diKbD8cIGC

“Gene therapy will be the only thing that may save people’s lives. If we can do this and spread it around the globe, that will be big for the rare disease community” -Peter Marks, M.D., Ph.D. @US_FDA #RDDS #DrugDevelopment

$SRPT Just the video link

Congratulations to Faye Leijenhorst who is the winner of the 2023 Girdie Limelight Design Contest! 💚 We want to thank everyone who submitted a design for the contest. The LGMD community had a lot of creative talent to share #LgmdAwareness #GirdieLimelight #TogetherWeAreStronger

Check out the latest "LGMD Spotlight Interview" featuring Peter who has LGMD 2C/R5 and lives in the United States. 💚 Read the full interview at https://t.co/u4Ld6waEZe #CureLGMD #LGMDawareness #LGMD2C #LGMDR5

Join us for #MDA Virtual Learning Series: Limb Girdle #MuscularDystrophy (#LGMD). This new disease-specific virtual learning event will take place over 5 weeks, starting on Wednesday, 4/19 at 6:00pm ET for updates, information & resources specific to LGMD: https://t.co/fuuNUF4DXH