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Hunter syndrome

Sled dogs racing toward finish
Biotech

Denali rare disease drug clears safety study, reduces biomarkers

Denali's drug met its primary safety endpoints and reduced key biomarkers of Hunter syndrome in a phase 1/2 trial, the biotech announced.
Darren Incorvaia Feb 6, 2025 12:05pm
Graphic of two hands shaking under a red and purple light

RegenXBio sells Hunter syndrome gene therapy commercial rights

Jan 14, 2025 5:56am
packing boxes moving layoffs failure shut down

Amid pivot, Denali Therapeutics sends staff packing

Feb 26, 2024 12:22pm
pedal to metal fast car speed gas

RegenXBio gets FDA support for accelerated gene therapy filing

Aug 3, 2022 10:00am
Takeda HQ

Takeda scraps dream of getting rare disease drug to market

May 11, 2022 8:30am

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