Despite the obstacles, Riley Herrera had a life filled with family, sports, travel, faith and hope before his death on Nov.

Cumberland Pharmaceuticals’ Duchenne muscular dystrophy (DMD) drug has improved the amount of blood pumped by the heart, ...

The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years ...

Percheron Therapeutics says it will abandon its lead drug candidate altogether, following December's Duchenne muscular dystrophy trial flop.

Some of the market’s biggest winners start off as the smallest players. While trillion-dollar tech giants like the ...

Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX) shares began Tuesday sharply higher. Cumberland, a specialty pharmaceutical company with development efforts focused on new products for rare diseases, ...

A seven-year-old boy from Dublin is on his way to California to undergo testing after being diagnosed with a rare condition last year.

A newly discovered line of communication between body muscle and the pituitary gland may play an unexpected role in female fertility, according to a new study. McGill University researchers found that ...

Fulcrum Therapeutics' promising treatment for Sickle Cell Disease and their strong financial position, despite setbacks.

After all the Tuberculosis outbreaks in Kansas, both the Lubbock Health Department and Covenant Medical Center explained what ...

While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

Muscular dystrophy is diagnosed using several tests. These may include: Muscle biopsy. A small piece of muscle tissue is removed and examined to confirm the diagnosis or rule out another muscle ...