BREAKING: Today, we announced the
@US_FDA
approved our therapy to treat Duchenne muscular dystrophy, providing a new treatment option for this rare, degenerative disease. Learn more about this important milestone here:
Are you attending
@CureDuchenne
FUTURES and have a question for Sarepta? Our team is available throughout the conference at our two booths and our Lego activity. Hope to see you there!
We’re thrilled to be a Diamond sponsor
@CureDuchenne
’s FUTURES conference on May 23-26 and look forward to connecting with families in Orlando, Florida.
Congratulations to Dr. Jerry Mendell on being named to the inaugural Time100 Health list! The list recognizes individuals who most influenced global health this year, and it is an acknowledgement of Dr. Mendell’s impact, innovation and achievement.
Today, we announced the opening of the 2024 Sarepta LGMD Grant Award Program. Open to non-governmental and patient advocacy organizations from across the globe, it aims to help shorten the LGMD diagnostic journey. Read our news release to learn more:
Today, we announced the opening of Route 79, The Duchenne Scholarship Program. Created to help those affected by
#Duchenne
pursue their post-high-school educational goals, the program will award 25 scholarships. Read the press release to learn more:
Leading up to
#RareDiseaseDay
, we’re talking about the importance of
#newbornscreening
for rare genetic diseases. We support the inclusion of Duchenne in existing
#NBS
programs so that that all patients have the chance to benefit from early diagnosis and treatment.
The inclusion of Duchenne on Minnesota's newborn screening panel is a significant step forward. Congratulations to the advocates who supported this cause and kudos to the Advisory Committee for recognizing the urgent need. Because when it comes to Duchenne, time is muscle.
NEWS: Today we announced that screening is underway in Study SRP-9003-301, also known as EMERGENE, a Phase 3, multi-national, open-label study of investigational candidate SRP-9003 for the treatment of LGMD Type 2E. Read the press release:
Today, we announced topline results from EMBARK, a double-blind placebo-controlled, Phase 3 clinical study of our gene therapy in patients with Duchenne muscular dystrophy.
Thank you, Assemblyman Hevesi, for your steadfast leadership on this important issue and for making a difference in the lives of Duchenne patients born in New York.
I am extremely proud that my bill
#A5042
was signed into law. This will ensure that all newborns are screened for
#DuchenneMuscularDystrophy
, allowing medical providers to make early detections, mitigate impacts and save lives.
What’s in a gene therapy dose? It depends. How much gene therapy is needed depends on the nature of the disease being treated, and how many and what type of cells are affected. This video takes you inside the science to explore gene therapy and gene therapy dosing. Watch: