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BioMarin will acquire Alesta Therapeutics for $275 million upfront, bolstering BioMarin's unit focused on rare skeletal conditions.

The combination regimen significantly extended the time patients lived without their melanoma returning, compared with Keytruda alone.

Hims & Hers CEO Andrew Dudum spoke to CNBC about the FTC's lawsuit against the company, compounded GLP-1 drugs and AI.

Nome uses AI and drug-development expertise to help rare disease families turn genetic diagnoses into potential paths toward treatment.

A U.S. appeals court has ruled in favor of Gilead Sciences against companies known as alternative funding programs that import medicines from overseas.

Novo Nordisk CEO defended the company's decision to sue rival Eli Lilly over its weight-loss drug advertising.

Federal dollars in the two health insurance programs would not go toward gender-affirming care, but states could still cover the treatments.

The quarter wasn't totally clean, but strong profitability matters the most.

Doctors and other experts say that splitting the measles, mumps and rubella shot has no scientific basis and is unlikely to get off the ground.

Jazz Pharmaceuticals will acquire Actio Biosciences, Inc., a deal that expands Jazz's pipeline and presence in rare epilepsy treatments.