Despite a long-held reputation as a relatively mild party drug, nitrous oxide is becoming increasingly dangerous and building ...
The Food and Drug Administration aims to evaluate treatments for rare diseases based on plausible evidence that they would work — without requiring a clinical trial first.
How innovative technologies like AI and gene editing are advancing rare disease drug discovery and development.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced the U.S. Food and Drug Administration (FDA or the Agency) has ...
Some tuberculosis program managers report double-digit increases, and the cost of antibiotics to treat it is rising.
Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big ...
PharmAla Biotech Holdings Inc. (CSE: MDMA)(OTCQB: MDXXF), a biotechnology company focused on the research, development, and ...
New Program Designed as Platform-based Approach for Direct Correction of Mutations Causing PKU; Investigational New Drug (IND) Filing for ...
VTVT stock outlook: underfollowed T1D catalyst with Phase 3 CATT1 cadisegliatin data due 2H2026, improved timeline and runway ...
Preeclampsia is a serious, potentially fatal high blood pressure disorder that can develop during or after pregnancy. It affects between 5% and 8% of pregnancies.
In Johnson County, Iowa, the number of tuberculosis cases has increased in recent years — and so has the cost of containing it. The cost of contact tracing and surveillance, traveling each day to ...
PDUFA action date set for August 23, 2026 If approved, DTX401 will be the first treatment to address the underlying cause of GDSIa NOVATO, Calif., Feb. 23, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmace ...