-
Duchenne Added to Minnesota’s Newborn Screening Panel
PPMD is excited to announce another significant milestone: Minnesota has officially approved the addition of […]
-
Sometimes living with Duchenne is simply too hard
I love to read, but finding time to sit down with a good book can […]
-
CHMP Issues Negative Opinion for Renewal of Conditional Marketing Authorization for Translarna™ (ataluren) in Europe
PPMD is disappointed to learn that the European Medicines Agency (EMA)’s Committee for Medicinal Products […]
-
Coping with seasonal affective disorder and FSHD as best I can
The symptoms of my facioscapulohumeral muscular dystrophy (FSHD) make staying positive a daily challenge. Many […]
-
PepGen Announces First Patient Dosed in CONNECT1-EDO51 Phase 2 Clinical Trial of PGN-EDO51 for Duchenne Muscular Dystrophy Patients Amenable to Exon 51 Skipping
As an early funder of PepGen, we are pleased to share that PenGen has dosed […]
-
Santhera Receives Approval for AGAMREE® (Vamorolone) as a Treatment for Duchenne Muscular Dystrophy in the United Kingdom
AGAMREE (Vamorolone), whose development at ReveraGen Biopharma was supported by CureDuchenne, has been approved in […]
-
Introducing PPMD’s Gene Therapy Hub
We are excited to announce the launch of PPMD’s Gene Therapy Hub! Gene therapy has […]
-
Join us at PPMD’s 30th year meetings and events
Happy New Year! As PPMD celebrates its 30th anniversary in 2024, we’re thrilled to announce […]
-
Juvena’s JUV-161 named FDA orphan drug for DM1
The U.S. Food and Drug Administration (FDA) has given orphan drug designation to JUV-161, Juvena Therapeutics’ lead […]
-
PTC Therapeutics – EMFLAZA® LANDSCAPE IN 2024
Join PTC Therapeutics and CureDuchenne for this prerecorded webinar to hear important information regarding Emflaza, PTC Cares and […]
