A renewal of the FDA’s pediatric rare disease voucher program is couched within a massive $1.2 trillion spending bill the ...
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Genespire to take rare disease gene therapy to clinic in 2026
GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia.
Backed with $82 million and led by two of BioMarin’s former executives, Mendra plans to acquire rare disease assets and use ...
Dr Sudheendra Rao N R from the Organization for Rare Diseases India reveals that approximately 70 million are affected by ...
Mendra, Inc. ("Mendra"), a biopharmaceutical company built to advance promising therapies for rare disease medicines, utilizing artificial intelligence (AI), today announced its launch in conjunction ...
Danish dermatology specialist Leo Pharma and its CEO Christophe Bourdon came to San Francisco to attend the J.P. Morgan ...
Jaguar has received the initial $16M payment related to the company's recently executed US out-license agreement for Mytesi ® ...
A 2-year-old boy died from a rare disease with flu-like symptoms, just weeks before his third birthday. Hudson Hughie Martin, ...
Torie Bosch is the First Opinion editor at STAT. Celena Lozano’s son Benny, who turned 5 in November, loves trains, trucks — anything that goes. He also has a rare disease, PURA syndrome. Earlier this ...
The Priority Review Voucher (PRV) program is designed to incentivize rare disease drug development.
After years stuck in the “doldrums,” the biopharma sector is in a “very good place” heading into the new year, analysts told ...
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