From burdensome weekly Elaprase infusions to single‐dose gene therapy, the Hunter Syndrome pipeline is racing to rewrite outcomes-and expectations. When a rare disease finally attracts true CNS ...

DelveInsight’s, “Hunter Syndrome Pipeline Insight 2025” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in Hunter Syndrome pipeline landscape. It covers the ...

Most cases of the syndrome occur sporadically, meaning the genetic changes behind the condition appear randomly in early development rather than being passed down through generations. Only in rare ...

for the treatment of mucopolysaccharidosis II (MPS II), also known as Hunter syndrome. MPS II is a rare, X-linked recessive disorder caused by lysosomal iduronate-2-sulfatase (I2S) enzyme ...

"This regulatory milestone brings us closer to our goal of delivering a new treatment option to the Hunter syndrome community as we continue to listen, learn, and seek their advice in bringing ...

Tividenofusp alfa is designed to address both cognitive and physical symptoms of Hunter syndrome, a rare genetic disorder primarily affecting males worldwide. The current standard of care does not ...

A Denali Therapeutics drug for the rare enzyme deficiency Hunter syndrome is still in pivotal testing, but the company has guidance from the FDA on a pathway to get this therapy to the market sooner.

Hunter syndrome, also called mucopolysaccharidosis type II (MPS II), is characterized by patients' inability to break down certain sugars, which leads to tissue and organ damage that ultimately ...

GC Biopharma announced on the 5th that the Ministry of Food and Drug Safety has designated the treatment for severe Hunter syndrome, 'Hunterase ICV (intracerebroventricular),' as an orphan drug.

Fifteen-year-old Dominic Henriquez from Prosper, Texas, is bringing hope to boys like him who live with Hunter syndrome, a rare genetic disorder that primarily affects males. Dominic’s journey ...

DNL310 received Breakthrough Therapy Designation; BLA filing for Hunter syndrome is anticipated in early 2025. Denali's $1.28 billion cash runway supports expansion efforts, aiming for ...