Rare Disease Report
Patients & Caregivers


Revance Therapeutics’ lead drug candidate DaxibotulinumtoxinA for Injection (RT002) has been granted orphan drug designation by the FDA to treat cervical dystonia.
Tremeau Pharmaceuticals, Inc. was granted orphan drug designation this morning for TRM-201 (rofecoxib), intended for the treatment of hemophilic arthropathy.
The FDA granted Breakthrough Therapy Designation and Orphan Drug Designation to PellePharm for its compound topical patidegib for use in patients with Basal-cell nevus syndrome (BCNNS).
Alfred George, Jr., M.D. of Northwestern University summarizes presentations on ATP1A3 in Japan in September
Rare Disease Report sat down with Timothy Miller, Ph.D., President and CEO of Abeona, to discuss the excitement surrounding ABO-102.
The 2017 Congenital Muscular Dystrophy (CMD) Scientific and Family Conference made for the largest-ever gathering of CMD-affected individuals, family members, and experts.
Alynylam has initiated submission of a rolling NDA to the U.S. FDA.
The U.S. FDA approved Mepsevii (vestronidase alfa-vjbk) for use in pediatric and adult patients with mucopolysaccharidosis type VII (MPS VII), often referred to as Sly syndrome.
This morning, Akcea announced that all its marketing applications for volanesorsen have been accepted for review in the United States, Europe and Canada.
Pro Football Hall of Famer Jim Kelly speaks about the death of his only son to Krabbe Leukodystrophy, and why it changed his life for the better.
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