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~ Supraphysiological expression of α-Gal A activity maintained for over a year in longest treated patient as of data cutoff date ~ ~ Stable Lyso-Gb3 levels maintained post-dosing, regardless of...

PRINCETON, N.J., Feb. 03, 2026 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced the presentation of new data from clinical and real-world studies of Galafold® (migalastat) in...

Sangamo announces it has begun a rolling submission of its BLA to the FDA for , an investigational gene therapy for adults with Fabry disease

~ All patients in the first cohort achieved between 27- to 208-fold increases in α-Gal A activity relative to mean normal level ~ ~ All patients in first cohort discontinued enzyme replacement...

New York, USA, June 10, 2025 (GLOBE NEWSWIRE) -- Fabry Disease Market to Grow Rapidly During the Study Period (2020–2034) | DelveInsight  The Fabry disease market is growing due to rising...

New York, USA, May 19, 2025 (GLOBE NEWSWIRE) -- Lysosomal Storage Disorders Market Set to Expand Across the 7MM During the Forecast Period (2025–2034) with Advances in Enzyme Replacement Therapies |...

~ Independent Data Monitoring Committee recommends proceeding with dosing of second cohort after planned safety assessment ~ ~ Company expects to initiate enrollment of second dose cohort in the...

LEXINGTON, Mass. and AMSTERDAM, Sept. 23, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical...

LEXINGTON, Mass. and AMSTERDAM, Aug. 15, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical...

LEXINGTON, Mass. and AMSTERDAM, Nov. 29, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical...