Rare Disease Report
Patients & Caregivers


Chef Paul Wahlberg, Dr Monaco of Tufts, Tim Boyd of NORD among those helping for World Duchenne Awareness Day in Boston
A robotic exoskeleton has been created by researchers from the National Institute of Health (NIH) to assist patients with cerebral palsy.
Drs. Mendell, Sahenk, and Rodino-Klapac explain why Exondys-51 (etepelrisen) got approved and Kyndrisa (drisapersen) did not.
The movie, based on the best-selling book, stars Julia Roberts and Owen Wilson as the parents of mandibulofacial dysostosis patient Auggie.
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.
If their DMD drug gets approved, Capricor Therapeutics would receive a Priority Review Voucher that is worth millions of dollars.
The FDA has granted Orphan Drug Designation to Mallinckrodt for their potential Duchenne muscular dystrophy (DMD) treatment, MNK-1411.
On Friday afternoon at the PPMD Patient Connect Meeting, Catabasis, Mitobridge, and Fibrogen provided clinical data updates.
DMD updates from Pfizer, BMS, Capricor,Reveragen, Santhera, Italfarmaco, Akashi at PPMD's patient conference.
A who's who from the pharma industry will be at the DMD conference to share clinical trial updates
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