So much has changed in the Duchenne research landscape since we were founded back in 1994, but your commitment has been a constant since our story began.
You’ve never stopped fighting, and the promise for our community has never been greater, with recent breakthroughs in gene therapy,…
What an odd year it has been. I won’t say it has been a bad year, not entirely, but it has been dark. And I don’t think it’s just me.
Even social media, something I turn to for connection, a familiar friend, a warm embrace…has felt toxic. So many Facebook…
Yesterday, the New England Journal of Medicine (NEJM) published “Single-Dose Gene-Replacement Therapy for Spinal Muscle Atrophy.”
Spinal muscular atrophy…Continue