The big pharma company will no longer progress emugrobart to late-stage trials in FSHD and SMA due to a lack of efficacy.

Mirecule, Inc., a leader in Antibody RNA Conjugate (ARC) therapeutics targeting rare neuromuscular diseases, today announced an expansion of its strategic collaboration with Sanofi. This collaboration ...

Roche (RHHBY) (RHHBF) has reiterated its plans to continue clinical trials for its anti-myostatin antibody emugrobart (GYM329/RO7204239) in obesity despite mid-stage trial setbacks for the candidate ...

An experimental RNA therapy from Avidity Biosciences has early clinical trial results showing it reduced by half the expression of a gene at the root of a rare, inherited form of muscular dystrophy ...

Avidity Biosciences said Wednesday morning that an experimental medicine successfully knocked down the gene behind a form of muscular dystrophy being pursued by a fleet of drug companies. The ...

FSHD Society to host Avidity webinar on positive initial data from the Phase 1/2 FORTITUDE™ trial for people living with FSHD today at 1:00 p.m. ET "This World FSHD Day, we are excited to be joining ...

VANCOUVER, British Columbia--(BUSINESS WIRE)--SOLVE FSHD, a venture philanthropy organization dedicated to catalyzing innovation and overcoming barriers to accelerate new therapies for ...

Facioscapulohumeral muscular dystrophy is a rare genetic disorder that causes progressive muscle weakness and atrophy, predominantly in the face, shoulders, and upper arms, in an estimated 1 in 8,000 ...

Mark J. Christman doesn’t have the strength to hold his nearly 8-month-old grandson, William “Willie” Chavara. “I am weaker every year,” said Christman, who was diagnosed in 1977 with ...

The investigational drug losmapimod did not significantly change expression of the gene behind facioscapulohumeral muscular dystrophy (FSHD), but it was associated with potential improvements in ...