U.S. congressional appropriators continue to march to a different drumbeat than the cadence President Donald Trump directed for the NIH and FDA.

As investors waited Wednesday afternoon for Sarepta Therapeutics Inc.'s second-quarter earnings, news of a managed access program (MAP) with Clinigen Group plc's Idis division for Exondys 51 (eteplirsen) in Duchenne muscular dystrophy (DMD) had Wall Street speculating that the deal would add to fiscal-year 2017 guidance of more than $95 million.

DUBLIN – Horama SA, an early stage French gene therapy firm, is waiting for regulatory feedback on its first clinical trial authorization (CTA) before moving into clinical development.

The House Ways and Means Committee met July 19 to examine the government's efforts to tamp down on Medicare fraud, but legislation is in the works that would increase the penalties for Medicare fraud even as the Department of Justice (DoJ) announced a series of arrests related to more than $1.3 billion in Medicare fraud.

Medical device sales growth beat out that for pharmaceuticals in the latest quarter from Johnson & Johnson. That comes scarcely a year and a half after the conglomerate announced a restructuring of its devices business in the face of non-existent growth. Key to the boost has been acquisitions, which drove much of this quarter's gains.

Treating animals with a drug that inhibited the cellular integrated stress response could reverse memory deficits caused by traumatic brain injury (TBI), even if treatment was delayed for several weeks after the injury.

Cymabay Therapeutics Inc., of Newark, Calif., said it priced its underwritten public offering of 13 million shares of its common stock at $6.50 each.

Axovant Sciences GmbH, of Basel, Switzerland, reported data from a preclinical study in a poster presentation at the 2017 Alzheimer's Association International Conference in London.

Tiziana Life Sciences plc, of London, said it enrolled the first patient in its phase IIa trial testing milciclib, a CDK inhibitor, in patients with refractory hepatocellular carcinoma (HCC).

Brainstorm Cell Therapeutics Inc., of Hackensack, N.J., said it signed definitive agreements with Massachusetts General Hospital and California Pacific Medical Center to enroll patients in the planned phase III trial of stem cell therapy candidate Nurown in amyotrophic lateral sclerosis, pending FDA and institutional review board approvals.