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Rare Diseases

Cases of monkeypox are being investigated in Europe, the United States, Canada, and Australia

Cases of monkeypox are being investigated in several European countries as well as the US, Canada and Australia, according to health authorities and local...

FDA lifts ban on LogicBio’s clinical trial on pediatric patients with methylmalonic acidemia

LogicBio Therapeutics, Inc., a clinical-stage company advancing a diversified pipeline of genetic medicines addressing rare disorders from infancy through adulthood, May 9th announced that...

Novo Nordisk raises 2022 outlook after strong quarter, shares up

Novo Nordisk targets higher sales and operating profit for the year on the back of projected profit growth in the first quarter thanks to...

FDA Approves alpelisib as first and only treatment for PROS, a spectrum of rare conditions

Novartis on Wednesday announced that the U.S. Food and Drug Administration (FDA) granted accelerated approval to Vijoice® (alpelisib) for the treatment of adult and...

Sanofi’s rare disease drug gets world’s first approval in Japan

The Japanese Ministry of Health, Labor, and Welfare (MHLW) has granted marketing authorization for Xenpozyme® (olipudase alfa) for the treatment of adult and pediatric...

U.S. FDA approves drug to treat seizures associated with Lennox-Gastaut Syndrome, a rare form of childhood epilepsy

UCB, a global biopharmaceutical company, on Monday announced that FINTEPLA (fenfluramine) oral solution CIV has been approved in the United States, by the U.S....
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