Following the first-ever treatment for spinal muscular atrophy in the womb, physicians say a 2-year-old girl shows no signs ...

The Montana House of Representatives on Wednesday voted overwhelmingly against House Bill 371, a measure that would have banned mRNA vaccines — used primarily in COVID-19 vaccines and booster shots — ...

Company to submit sNDA around the end of 1Q 2025 seeking traditional approval of FILSPARI® (sparsentan) for FSGSNet product sales of FILSPARI ...

Chimerix holds the potential to have the first approval of dordaviprone for treating recurrent H3 K27M mutant high-grade ...

YORVIPATH® (palopegteriparatide) is now registered by the Therapeutic Goods Administration (TGA) for the treatment of chronic hypoparathyroidism in adults1First-in-class novel PTH replacement therapyA ...

A panel of experts that advises the U.S. Centers for Disease Control and Prevention on vaccine policy will not meet as previously scheduled next week.

BioMarin's gene therapy pipeline has had setbacks, including a three-year delay in US approval of Roctavian and stalled development of PKU gene therapy BMN 307 as the Food and Drug Administration ...

Last week, a Texas judge ordered Dr. Margaret Carpenter — a New York abortion doctor — to pay at least $100,000 in penalties ...

BridgeBio’s ATTR cardiomyopathy drug, Attruby, achieved promising sales numbers in the weeks after its FDA approval.

"Any place that gets cut, it's going to have an impact, because there's not any spare personnel at FDA,” said former agency ...

A toddler was successfully treated for a rare genetic disease, spinal muscular atrophy, after world-first in-womb therapy.

BridgeBio's launch of Attruby in the U.S. is off to a strong start. Read how this successful effort puts the biotech in a ...