The disease gradually takes away the skills children have learned—leading to seizures, movement difficulties and profound ...
Denali Therapeutics faces an FDA review delay for its Hunter syndrome drug, pushing potential approval of tividenofusp alfa to April 2026.
KNOXVILLE, Tenn. — Bryan and Blair Begbie said all they want for Rowan — their youngest daughter — is the chance to be a kid. However, the Food and Drug Administration issued what is called a Complete ...
JCR and MEDIPAL announced that the Ministry of Health, Labour and Welfare of Japan has granted orphan drug designation to JR-446 for MPS IIIB.
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Spruce Biosciences (SPRB) Stock Jumps 30% After Hours Following Red Hot Rally On Monday: Here's Why
Spruce Biosciences shares soared after the FDA granted Breakthrough Therapy Designation to its lead treatment for Sanfilippo Syndrome Type B.
Investing.com -- Spruce Biosciences (NASDAQ:SPRB) stock surged 60% after the company announced it received Breakthrough Therapy Designation from the U.S. Food and Drug Administration for its ...
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Parents of children with rare genetic condition concerned about recent pharmaceutical delays
The Begbies say they don't want Sanfilippo Syndrome to take away happy moments from their daughter, but new regulations have made it hard to receive proper care. Orcas attack and sink another boat off ...
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