Proud to collaborate on this piece led by @oabdulhamid and @pandaneuro. Decision to pursue Elevidys treatment requires individualized detailed counseling and consideration of potential risks / benefits given unknowns.

PPMD is excited to share that FDA has made the decision to expand the labeled indication for ELEVIDYS, a microdystrophin #genetherapy used to treat #Duchenne, to include individuals at least 4 years of age and remove ambulation restrictions. Learn more:

BREAKING NEWS: @Sarepta Therapeutics Announces Expanded US #FDA Approval of #ELEVIDYS to #Duchenne Muscular Dystrophy Patients Ages 4 and up. MORE DETAILS TO COME WITH WEBINAR INVITE. STAY TUNED.

Last week, I had a PET scan to look into my Castleman disease, and also to determine whether I’d had a recurrence in my liver cancer.   Great news, the scan came back completely clear and I’m so thankful for that.   But it also reminds me that we have so much work to do and at

PepGen is honored to share stories of the incredible members of the neuromuscular disorder community living with DM1 and DMD. To learn more about Colin’s story, who is living with DMD, visit: https://t.co/iCfyGQFU9A

Pat Furlong and Buddy Cassidy spoke to UN diplomats about recognizing September 7 as World Duchenne Awareness Day. WDO is grateful for this initiative and is happy so many of our members responded to the request to send a letter to their permanent UN Representative.

Jaxson just became the FIRST patient in Florida to receive the groundbreaking ELEVIDYS gene therapy treatment for Duchenne Muscular Dystrophy! NCH Florida is one of only 3 hospitals in the state to receive this therapy. 🎉💙💪 Follow his journey: https://t.co/K5oToqnvoR

Will be an interesting talk!

➡️ How strong is your knowledge of the application and potential complications of using adeno-associated virus gene therapies for neuromuscular disorders? #AAV #MedEd #CME #NeuroTwitter 📍 https://t.co/t9DWHuQgb1

...here is Julie's and Kim's blog post on #STX1A and #STX1B in neurodevelopmental disorders ...both genetic etiologies are increasingly identified causes of neurodevelopmental disorders ...however, the phenotype for these conditions is different from other SNAREopathies.

Looking for some 🧠CME?! Directed by two incredible Neurologists (Sashank Prasad, MD and Tammy Kaplan, MD) Neurology for the Non-Neurologist June 19 - 21, 2023 Virtual format w/ lectures, case-based discussion, & live Q&A! CME for MDs, APPs, NPs, RNS https://t.co/bynWUahoAp

Neurology® Video Journal Club's newest series features topics in: #MyastheniaGravis, #ALS, and neuromuscular disease. Start watching now: https://t.co/81E8LX6NLl

New #MolTherapyNucAcids paper: Partial restoration of brain dystrophin by tricyclo-DNA antisense oligonucleotides alleviates emotional deficits in mdx52 mice.

We look forward to presenting at the virtual Stifel 2023 CNS Days. Tune in to the live webcast on Tuesday, March 28 at 2 pm ET. https://t.co/ufpyFaZQ2H

It's CACNA1A Awareness Day 💙💜💙💜 CACNA1A is a very large gene on the 19th chromosome. Variations in this gene can cause neurodegenerative disease and a variety of neurological symptoms. Learn more at https://t.co/3KhirAIS7o

It’s KCNQ2 Awareness Week! Nearly all of those affected experience seizures in the first several days of life. This is most often the symptom leading to testing resulting in a diagnosis of KCNQ2. https://t.co/T5se2z0ouU https://t.co/EnMSgjpc9b

The genetics of FIRES and status epilepticus in 2023 | Beyond the Ion Channel https://t.co/AED1VCEn2I @IlaeWeb @ChildNeuroSoc @AmEpilepsySoc

NEWS: SRPT today announced initiation and first patient dosed in the study known as VOYAGENE, a Ph 1 study of our investigational gene therapy for the treatment of LGMD Type 2E. Read the press release: https://t.co/udKdryArZH