Patients & Caregivers
RT002 Granted FDA Orphan Drug Designation
Revance Therapeutics’ lead drug candidate DaxibotulinumtoxinA for Injection (RT002) has been granted orphan drug designation by the FDA to treat cervical dystonia.
Hemophilic Artropathy Treatment Granted Orphan Drug Designation
Tremeau Pharmaceuticals, Inc. was granted orphan drug designation this morning for TRM-201 (rofecoxib), intended for the treatment of hemophilic arthropathy.
FDA Grants Breakthrough Therapy Designation to PellePharm for Gorlin Syndrome Treatment
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).
Report from the 6th ATP1A3 Meeting; Tokyo, Japan
Alfred George, Jr., M.D. of Northwestern University summarizes presentations on ATP1A3 in Japan in September
President and CEO of Abeona Explains the Excitement Surrounding ABO-102
Rare Disease Report
sat down with Timothy Miller, Ph.D., President and CEO of Abeona, to discuss the excitement surrounding ABO-102.
Highlights from the 2017 CMD Scientific and Family Conference
The 2017 Congenital Muscular Dystrophy (CMD) Scientific and Family Conference made for the largest-ever gathering of CMD-affected individuals, family members, and experts.
Alnylam Initiates Submission of NDA for hATTR Treatment
Alynylam has initiated submission of a rolling NDA to the U.S. FDA.
FDA Approves MPS VII Treatment
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.
Marketing Applications Accepted for FCS Treatment in US, Canada and Europe
This morning, Akcea announced that all its marketing applications for volanesorsen have been accepted for review in the United States, Europe and Canada.
A Journey of Perseverance Takes Jim Kelly Through the Death of His Son
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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