Jaguar has received the initial $16M payment related to the company's recently executed US out-license agreement for ...
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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.
A renewal of the FDA’s pediatric rare disease voucher program is couched within a massive $1.2 trillion spending bill the ...
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 ...
Backed with $82 million and led by two of BioMarin’s former executives, Mendra plans to acquire rare disease assets and use ...
Lozano is a rare disease mom, neuroscience Ph.D. candidate at UC Davis, and board member for the PURA Syndrome Foundation. In May, a historic moment in science and medicine was captured in a single ...
A 2-year-old boy died from a rare disease with flu-like symptoms, just weeks before his third birthday. Hudson Hughie Martin, ...
Danish dermatology specialist Leo Pharma and its CEO Christophe Bourdon came to San Francisco to attend the J.P. Morgan ...
The Priority Review Voucher (PRV) program is designed to incentivize rare disease drug development.
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 ...
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