The FDA has granted fast-track approval for a groundbreaking gene therapy indicated for a rare genetic disorder called aromatic L-amino acid decarboxylase (AADC) deficiency.

The U.S. patient population for Revuforj in its approved indication is roughly 2,000 according to Syndax. KMT2A leukemias ...

Moberg Pharma's MOB-015 has outstanding clinical trial data, 76% cure rate, double the current standard topical, Jublia. Read ...

J.P. Morgan analyst Anupam Rama has maintained their bullish stance on SNDX stock, giving a Buy rating on November 13. Anupam Rama’s ...

Ipsen to present new data on elafibranor at The Liver Meeting® 2024Launch of Iqirvo® (elafibranor)1 on track with expectations; encouraging feedback from healthcare providers ...

Put up against placebo in the phase 3 EMBARK trial, delandistrogene moxeparvovec (Elevidys) did not significantly improve ...

President-elect Trump’s decision to nominate Robert F. Kennedy Jr. for Health and Human Services (HHS) secretary will put some of the former independent presidential candidate’s most ...

Kennedy is Trump’s pick for health secretary, but he has a history of spreading misinformation about a range of issues.

Revumenib is now an FDA-approved treatment for adult and pediatric patients with relapsed/refractory KMT2A-rearranged acute ...

The device is in the early stages of approval by the US Food and Drug Administration and only a few have been implanted in people across the world.

Robert F. Kennedy Jr., an anti-vaccine activist and environmentalist, for years gained a loyal and fierce following with his ...