Newborn Screening for Duchenne Muscular Dystrophy

In a promising development, the Secretary’s Advisory Committee on Heritable Disorders in Newborns and Children (ACHNDC) has advanced the nomination of Duchenne muscular dystrophy (DMD) for inclusion in the Recommended Uniform Screening Panel (RUSP), a list of disorders that are screened for at birth.

Avidity exon skipping

Avidity Biosciences Announces Positive AOC 1044 Data

Avidity Biosciences, which received early funding from CureDuchenne, announced positive initial data from their EXPLORE44 Trial for individuals with Duchenne amenable to skipping exon 44.  Their experimental exon-skipping agent, AOC […]