FDA Approves Second Exon-Skipping Drug for Duchenne

The U.S. Food and Drug Administration has approved Vyondys 53 (golodirsen) to treat patients with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. Golodirsen is an antisense oligonucleotide that can ‘skip’ over aspects of the RNA to make person’s with...

FDA Puts Hold on Trials for Rare Bone Disease

The U.S. Food and Drug Administration has issued a partial clinical hold in two studies underway to test palovarotene chronic treatment of fibrodysplasia ossificans progressiva (FOP) and multiple osteochondromas, respectively. The partial clinical hold is aimed at the...

CRB1 Retinal Disease and Quality of Life

  Kristin Smedley, president of Curing Retinal Blindness Foundation, talks about what doctors need to know about conditions such as retinitis pigmentosa and Leber congenital amaurosis. Curing Retinal Blindness Foundation is a non-profit organization focused on...

Lipodystrophy is Often Misdiagnosed

  Andra Stratton, president of Lipodystrophy United explains how lipodystrophy is often misdiagnosed or under-diagnosed. Lipodystrophy is a rare medical condition in which persons have an abnormal distribution of fat in the body. There are numerous types of...
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