We are at a decisive moment where #Duchenne is just beginning to evolve from a death sentence to a manageable chronic condition. The U.S. FDA #acceleratedapproval pathway is key to seizing this moment and saving lives. More in STAT today.

$RHHBY $SRPT 'With this extensive experience in gene therapy, the hospital also aims at offering a novel and lifesaving gene therapy for another life-limiting neuromuscular condition, #Duchenne ' #GeneTherapy 💪 zawya.com/en/press-relea…

$SRPT I know nothing on this collaboration's status but from Rare Disease Day 2024: 'Genethon is responsible for commercializing the product GNT0004 in Europe (excluding the UK) and Sarepta is responsible for the rest of the world.' #Duchenne #GeneTherapy genethon.com/genethon-pursu…

Ohio is officially screening all newborns for #Duchenne #MuscularDystrophy , making it the first state in the nation to screen for #DMD ! #MDA will continue to advocate until all newborns are screened for DMD across the country. Join us: MDA.org/Advocacy #NewbornScreening

$SRPT #Duchenne #Elevidys #GeneTherapy #RareDisease Jason James Howard, MD Nemours Children's Health (Delaware): 💪youtu.be/n7-T1ZrDWsU?t=…

We are proud to be a Gold sponsor of the International Congress of Myology International Congress in Paris this week. Stop by our booth #11 to learn more about our trials in #Duchenne and #Becker #musculardystrophy .
myology2024.org

Fot's smile is travelling through his cheeks, to his eyes, to his entire body... that's not a normal smile anymore, it's a duchenne smile 🩷

Like #acceleratedapproval , #newbornscreening is one of the big structural entities we need aligned in our favor if we want to meaningfully pursue the next phase of progress against #Duchenne . So pleased to see Ohio and other states come online with screening.

$SRPT Wow. Just - wow. 💪 #Elevidys #GeneTherapy #Duchenne #RareDisease

Starting today, all newborns in the state of Ohio will be screened for Duchenne muscular dystrophy, which is typically not identified until 3-5 years old. Grateful for the decades of work and progress that led to this moment and to get to be a small part of the future!

Solid is a committed champion of newborn screening for Duchenne muscular dystrophy. We are proud to support Assembly Bill 2563 in the state of California and will continue our collaborative efforts to have Duchenne added to the national Recommended Uniform Screening Panel (RUSP).

If Taylor household could do weekends

Secure a last minute ticket to Cambridge away ✅

Derby collect said 3 points, all but securing promotion ✅

Run the London Marathon ✅

Raised over £1,800 for Duchenne UK ✅

#LondonMarathon #dcfc #duchenne

Mad few days but of to Dubai for treatment for my wee warrior .Duchenne Muscular Dystrophy came into our life not by choice but we will take on the world to try slow it down I won’t let DMD take our boy. Thankyou everyone for all your support 💚 some incredible people out there.

$SRPT #eteplirsen #Duchenne #RareDisease The exon-skipping drugs (ASOs): Jason James Howard, MD Nemours Children's Health 'I can tell you that there's a definite Improvement in their motor function; things like stair climbing and preservation of walking' 💪youtu.be/n7-T1ZrDWsU?t=…

Today we announced that the European Medicines Agency has granted Orphan Drug Designation for sevasemten (EDG-5506) for the treatment of Becker muscular dystrophy and for the treatment of Duchenne muscular dystrophy. Check out our press release:
investors.edgewisetx.com/news/default.a…

Em entrevista ao Bom Dia SP, falei sobre o Projeto de Lei 1063/2024, de minha autoria, que classifica a Síndrome de Duchenne como deficiência para todos os efeitos legais.

As pessoas com doenças raras e crônicas enfrentam desafios diários que exigem não apenas tratamentos

[PRESS RELEASE]
During the #Myology24 congress, the first clinical trial results of gene therapy (GNT0004) for Duchenne Muscular Dystrophy were presented today by Professor Francesco Muntoni, principal investigator of this trial, sponsored by GENETHON France
urlz.fr/qo5j

Join the GrowDMD study! Canadian youth, aged 12 to 25, affected by #Duchenne muscular dystrophy (DMD) and their parents or caregivers are invited to take part in an exciting research study.

To learn more or to participate in this project, please email [email protected]

Ohio will be the first state to screen all babies for #Duchenne ! More than 129,000 babies are born in Ohio every year, and the state expects to identify 35 babies with Duchenne annually. Learn more: parentprojectmd.org/ohio-to-begin-…

$SRPT $HNSA.ST SRP-9001-104 #imlifidase in DMD: • First high-level data read-out from Phase 1b (exp 2024) #Duchenne #Elevidys #GeneTherapy PDF: hansabiopharma.com/files/Financia…