Duchenne Progress Shows How the FDA Should Judge Orphan Drugs

A milestone in the treatment of Duchenne muscular dystrophy (DMD) passed recently with remarkably little public notice. That ...

Scientists apply genome editing to treat muscular dystrophy

KYOTO--Utilizing genome editing technology, a team of researchers has developed a much more effective treatment for a certain ...
The American Journal of Managed Care

Developments in Muscular Dystrophy Gene Therapy

Emma Ciafaloni, MD, FAAN, dives into the latest developments in gene therapy for muscular dystrophies, focusing on Duchenne muscular dystrophy, and discusses challenges, genetic causes, and the ...
WLWT

FDA expands approval of first gene therapy for rare form of muscular dystrophy

The Muscular Dystrophy Association trusts the decision of the FDA, which weighs the risks and benefits of the drug The Muscular Dystrophy Association trusts the decision of the FDA, which weighs the ...
Labroots

A New Approach in Gene Therapy for Muscular Dystrophy

Scientists have created a new gene therapy for Duchenne muscular dystrophy (DMD) that may not only help stop the disease in DMD patients, but might also help restore their damaged muscles in the ...

Sarepta's Duchenne gene therapy slows disease progression at three years

By Mariam Sunny Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy slowed disease ...
Yahoo

Second patient death reported with gene therapy for muscular dystrophy

WASHINGTON (AP) — Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in connection with its gene therapy for muscular dystrophy. Sarepta reported the ...
WTAE-TV

FDA expands approval of first gene therapy for rare form of muscular dystrophy

The U.S. Food and Drug Administration has given the green light for the first gene therapy that treats a rare form of muscular dystrophy to be used in most people who have the disease and a certain ...