The Japanese pharma had one asset rejected by the FDA and withdrew a regulatory application for another, but already this ...

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

Riding recent momentum in the Duchenne muscular dystrophy space, Capricor Therapeutics, Wave Life Sciences, Regenxbio and ...

Achondroplasia, which affects around one in 20,000 babies, has just one approved treatment: BioMarin’s Voxzogo. However, new investigational treatments are vying to compete in the area.

The settlement is the largest deal to date with the people primarily who played an “instrumental role” in driving the opioid ...

Robert F. Kennedy, Jr.’s recent disclosures have revealed several potential conflicts of interest, including investments in ...

There are currently no treatments available for celiac disease beyond a gluten-free diet. Several late-phase companies aim to ...

Less than a day into his second term, President Donald Trump ordered a freeze on communications at major public health ...

The San Diego–based company’s molecules avoid the well-trod GLP-1 pathway in favor of an alternate route in the gut.

The unsuccessful Phase III results are the latest to suggest that the blockbuster cancer drug is finally bumping up against ...

The drugs’ active ingredient, glatiramer acetate, has been linked to more than 80 cases of anaphylaxis worldwide since ...

Traditionally carrying a dire prognosis, the treatment paradigm for multiple myeloma is changing, with CAR T therapies, ...