The Inflation Reduction Act includes an exemption for orphan drugs for a single indication, but experts say this is far from sufficient to maintain momentum in the rare disease space.

The FDA had resolved the shortage of Wegovy in April, and telehealth providers were advised to stop selling compounded ...

Shares in both companies were down following news of the second death, casting fresh safety concerns over Elevidys.

Nicole Verdun, MD, super office director of the FDA’s Office of Therapeutic Products (OTP) and her deputy Rachael Anatol, PhD ...

Wall Street’s major market averages seesawed back and forth on Friday and finished mixed as Federal Reserve Governor ...

Notable research news last week included Sarepta Therapeutics reporting another death associated with a trial of its Duchenne ...

Sarepta shares face pressure after a second Elevidys-linked death and analyst downgrade over safety concerns and lowered revenue prospects.

The latest shake-up at the FDA forced out the top two officials in charge of reviewing cell and gene therapies, analysts said ...

Nicole Verdun, a highly respected individual in the cell and gene therapy space, was placed on administrative leave this week ...

The death of a boy with Duchenne muscular dystrophy, tied to a gene therapy, is above all a tragedy. But it is also ...

Detailed price information for Sarepta Therapeutics (SRPT-Q) from The Globe and Mail including charting and trades.