Handt is a writer, communications adviser, and founder of Charlie’s Cure. Maynard is a rare disease advocacy leader and founder of the Little Hercules Foundation. They say death is one of life’s few ...

Japanese drugmaker Nippon Shinyaku said on Monday that its Duchenne muscular dystrophy drug, Viltepso, part of a controversial class of treatments for the rare disease, had failed to reach its primary ...

Since NS Pharma launched its Duchenne muscular dystrophy (DMD) drug Viltepso (viltolarsen) in 2020, sales have steadily trended upward, with the company expecting the treatment to generate 20,100 ...

INDIANAPOLIS — A local mom is pushing for a rare and deadly disease to be added to Indiana's newborn screening panel. Duchenne muscular dystrophy affects at least 1 in 5,000 male births each year.

A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle degeneration. Researchers have discovered that an experimental compound ...

Disclosing new interim data from its Phase 1/2 AFFINITY DUCHENNE trial, Regenxbio (NASDAQ:RGNX) announced Thursday that its experimental gene therapy RGX-202 continued to benefit patients living with ...

Duchenne muscular dystrophy has several approved drugs, including a gene therapy that provides children who have the rare, inherited muscle-wasting disease the option of a one-time treatment. But each ...

Duchenne muscular dystrophy (DMD) is a genetic condition that causes progressive muscle weakness and wasting. It is an X-linked recessive disorder that occurs due to changes in the DMD gene, which ...

Duchenne muscular dystrophy (DMD) is a genetic disease that causes muscle weakness and motor skill difficulties due to the loss of muscle tissue from changes to dystrophin proteins. It mostly affects ...

Delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), showed high dystrophin expression and a favorable safety profile in young patients. Delandistrogene moxeparvovec ...

Over the past decade, in a quest to fight the deadly disease that ails his son, Manalapan’s Jim Raffone has trekked to Mount Everest base camp, built a record-setting Lego chain, appeared on a special ...

ANN ARBOR, Mich., Sept. 14, 2017 (GLOBE NEWSWIRE) -- Phrixus Pharmaceuticals, Inc. (“Phrixus”), a company focused on therapies for Duchenne muscular dystrophy (DMD) and heart failure, today announced ...