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
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
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