Jaguar has received the initial $16M payment related to the company's recently executed US out-license agreement for ...
A "gene silencer" (technically known as small interfering RNA, or siRNA), locally delivered by nanoparticles embedded in an ...
A renewal of the FDA’s pediatric rare disease voucher program is couched within a massive $1.2 trillion spending bill the ...
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 ...
GlobalData on MSN
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.
Dr Sudheendra Rao N R from the Organization for Rare Diseases India reveals that approximately 70 million are affected by ...
Backed with $82 million and led by two of BioMarin’s former executives, Mendra plans to acquire rare disease assets and use ...
The FDA’s rare pediatric disease priority review voucher program missed reauthorization at the last minute in 2024; advocates ...
Danish dermatology specialist Leo Pharma and its CEO Christophe Bourdon came to San Francisco to attend the J.P. Morgan ...
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, ...
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