Sarepta Therapeutics (SRPT) stock in focus as report says company hired a Trump-linked lobbying firm after deaths linked to ...

A 26-year-old Downriver man seen at Children’s Hospital of Michigan in Detroit since infancy for a rare bleeding disorder in ...

Connecticut is now participating in a new federal program aimed at expanding treatment access for patients with sickle cell ...

Ocugen says it is on track to reshape the market for gene therapies against eye disorders over the next three years, by ...

At only 6 months, Gerard and Kaitlin Norton’s daughter Madeline was diagnosed with PKAN, a rare neurodegenerative disorder ...

Sarepta Therapeutics hired a Trump-connected lobbying firm after the death of a teenage boy treated with its Duchenne ...

In this week’s edition of InnovationRx, we look at a biotech entrepreneur treating a rare genetic disease, new tech aimed at ...

From alveolar injury to extracellular matrix accumulation, the complex biology of pulmonary fibrosis is being unraveled ...

Novo Nordisk will advance its acquired ATTR amyloidosis with cardiomyopathy monoclonal antibody (mAb) to Phase III trials.

Excellos brings its donor-to-dose capabilities—including donor management, apheresis, cell characterization, and GMP ...

Six Queen’s researchers have been awarded a total of $5.8M from the Canadian Institutes of Health Research to advance projects aimed at improving health outcomes.

Key Points Non-GAAP earnings per share reached $0.74, surpassing estimates by $0.03 (non-GAAP), but GAAP revenue of $181.1 million missed expectations and rose 1.7% (GAAP) compared to the second ...