Patients & Caregivers
Boston Seeing Red on Sept 7th for Duchenne Awareness Day
Chef Paul Wahlberg, Dr Monaco of Tufts, Tim Boyd of NORD among those helping for World Duchenne Awareness Day in Boston
Walking Made Easier for Cerebral Palsy Patients with Robotic Exoskeleton
A robotic exoskeleton has been created by researchers from the National Institute of Health (NIH) to assist patients with cerebral palsy.
Why Did Eteplirsen Get Approved for DMD but Drisapersen Did Not?
Drs. Mendell, Sahenk, and Rodino-Klapac explain why Exondys-51 (etepelrisen) got approved and Kyndrisa (drisapersen) did not.
Wonder: Will The Movie Live up to Expectations Set by the Book?
The movie, based on the best-selling book, stars Julia Roberts and Owen Wilson as the parents of mandibulofacial dysostosis patient Auggie.
ALS Patient Continues to Race 3 Years After Diagnosis
Andrea Peet, ALS Patient, has been living with the rare condition for three years, but still competes in triathlons with help from her Team Drea Foundation.
Capricor Receives Rare Pediatric Disease Designation for DMD Drug
If their DMD drug gets approved, Capricor Therapeutics would receive a Priority Review Voucher that is worth millions of dollars.
MNK-1411 Gets Orphan Drug Designation for Treatment of DMD
The FDA has granted Orphan Drug Designation to Mallinckrodt for their potential Duchenne muscular dystrophy (DMD) treatment, MNK-1411.
DMD Clinical Trial Update at PPMD Patient Connect Meeting — Part 2
On Friday afternoon at the PPMD Patient Connect Meeting, Catabasis, Mitobridge, and Fibrogen provided clinical data updates.
DMD Clinical Trial Update at PPMD Patient Connect Meeting — Part 1
DMD updates from Pfizer, BMS, Capricor,Reveragen, Santhera, Italfarmaco, Akashi at PPMD's patient conference.
DMD Conference Begins This Week
A who's who from the pharma industry will be at the DMD conference to share clinical trial updates
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