Avidity Biosciences, Inc. , a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCstm), today announced it is joining the ...

ITF, IntraBio and Orchard are among the companies that have won FDA nods in the past year for Duchenne muscular dystrophy, ...

University of Adelaide research into health and fertility has been funded in the latest round of the National Health and ...

Solid Biosciences reported recent positive data in developing their gene therapy candidate SGT-003. The phase 1/2 INSPIRE ...

Givinostat should be available to people with Duchenne muscular dystrophy through a type of compassionate use scheme.

The Muscular Dystrophy Association (MDA) announced today that Robert Califf, MD, MAAC, former Commissioner of the U.S. Food and Drug Administration and currently Instructor in Medicine at Duke ...

There is a broad consensus in the medical community in support of Duchenne newborn screening and we in Connecticut are hoping ...

In the phase 1/2 INSPIRE DUCHENNE trial, interim data showed an average microdystrophin expression of 110% among participants ...

In an international collaboration, researchers have made an important breakthrough in the therapeutic delivery of microRNAs ...

In honor of Rare Disease Day, here are 10 new treatments approved by the FDA to treat rare diseases over the past year.

Entrada Therapeutics is finally able to get its Duchenne muscular dystrophy candidate (DMD) back on track after the FDA ...