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- STAT+: FDA approves a new gene therapy for Sanfilippo syndrome, an ultra-rare diseaseThe FDA approved a new gene therapy for Sanfilippo syndrome type A, an ultra-rare disease sometimes known as childhood Alzheimer’s.STAT · 1mo ago↗ source
- FDA approves Ultragenyx's gene therapy for Sanfilippo syndrome type AThe FDA granted full approval to a rare disease gene therapy from Ultragenyx on Thursday, giving the company a much-needed boost in an otherwise difficult year. The therapy, called UX111, will be b…Endpoints · 1mo ago↗ source
- Two more biotechs, Marea and North Immunology, do reverse mergersThe reverse merger party is still buzzing. This time, it's a cardio and endocrine startup with two drugs in Phase 2 and a biotech working on a long-acting immunology and inflammation (I&I) drug. Mi…