#rare diseases

3 articles tagged with "rare diseases"

FDA Approval of Denali Medicine for Hunter Syndrome: Implications for Rare Disease Drug Development

The FDA's approval of Denali Medicine's treatment for Hunter syndrome indicates a pivotal moment in the regulatory landscape for rare disease therapies, amidst heightened scrutiny.

Editorial Staff 11 days ago

Life

FDA Approval of Denali Therapeutics Drug Enhances Rare Disease Treatment Landscape

The FDA's recent approval of Denali Therapeutics' drug for Hunter syndrome signifies a pivotal change in regulatory attitudes towards rare disease therapies.

Editorial Staff 12 days ago

Life

Sen. Johnson Questions FDA's Drug Approval Process for Rare Diseases

Senator Ron Johnson is scrutinizing the FDA's rejection of treatments for rare diseases, following a discussion with Commissioner Marty Makary that raised significant concerns.

Editorial Staff 27 days ago