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 ...

On March 10, 2026, from 12:00 p.m. -- 1:30 p.m. ET, in room Key West ABCD at the Hilton Orlando, Avidity will host an industry forum lunch titled "Biomarker Advancements in Rare Neuromuscular Disease: ...

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 ...

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 ...

Facioscapulohumeral muscular dystrophy (FSHD) is one of the most common forms of muscular dystrophy, and impacts almost one million people around the world. It is a genetic disorder that causes the ...

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 ...

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 ...

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 ...

SAN DIEGO, April 22, 2020 (GLOBE NEWSWIRE) -- Bionano Genomics, Inc. (Nasdaq: BNGO) announced that the University of Iowa Hospitals and Clinics (UIHC) will switch their method of clinical molecular ...

A 5K walk will be taking place this weekend in observance of a rare disease that many have never heard of. Joanne Dalessandro and her warriors will be in Barrington, Illinois, for the Chicagoland Walk ...