FDA unveils pathway ultra-rare disease therapies to approve gene-editing and RNA treatments when trials are unfeasible for small populations.

Plasmalogen precursors for Alzheimer’s are named as first program candidate There are nutrients and dietary compounds ...

At an event on Monday, the agency officially unveiled long-awaited draft guidance meant to help speed the development of bespoke treatments for extremely rare diseases.

Federal health officials on Monday laid out a proposal to spur development of customized treatments for patients with hard-to-treat diseases, including for rare genetic conditions that the ...

The FDA Modernization Act of 1997 established the Fast Track designation to expedite developing and reviewing new drugs designed to fill unmet medical needs and treat serious conditions. The FDA Fast ...

Psychedelic drug development can still benefit from the FDA Commissioner’s National Priority Review Voucher Pilot Program, though the CNPV designation was not given to Compass Pathways’ synthetic ...

The FDA has proposed new guidelines to facilitate the approval of customized treatments for rare diseases, aiming to address ...

In 2004, the U.S. Food and Drug Administration (FDA) issued its Botanical Drug Development Guidance, later revised in 2016, establishing a formal pathway for the development of regulated botanical ...

GlucaPharm is developing GEP44, a novel, patented peptide (similar in function to Ozempic, Wegovy, Mounjaro, but with reduced risk of adverse events) discovered by researchers at Syracuse University.V ...

Angiogenesis, the formation of new blood vessels, is crucial angiogenesis in tumor growth, proliferation, invasion, and metastasis. Advances in molecular biology have identified critical biomolecules ...