Analysis from continued follow-up of Phase 1/2 clinical trial data in Hunter syndrome (MPS II) reinforces potential for tividenofusp alfa ...
Data represents up to 8.5 years of follow-up and are consistent across age, dose, and genotypeBLA resubmitted to U.S. FDA in January 2026; ...
Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological disorders with significant unmet medical need ...
AMT-191 has been granted both Orphan Drug and Fast Track designation by the U.S. Food and Drug Administration.
PPT1 is a recombinant protein used to treat an ultra-rare disease called Batten disease CLN1 for which there is ...
Xanthine oxidase inhibitors, primarily allopurinol and febuxostat, are the mainstay of treatment. The goal of chronic gout management includes lowering serum urate levels to below 6 mg/dL. 5 Overall, ...
September 17, 2008 (Munich, Germany) — A new study has found that early enzyme-replacement therapy brings the most benefit in patients with Fabry's disease. Dr Frank Weidemann (University Hospital ...
Maharasthra BJP MP Anil Sukheorao Bonde on Friday urged the government to provide life-long and uninterrupted Enzyme Replacement Therapy to children suffering from rare diseases under the existing ...
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Why Everyone on TikTok Is Talking About DMK Enzyme Treatment—And What Dermatologists Think
I was scrolling through TikTok a couple of weeks back, when I suddenly came across a video that had me instantly intrigued. Outerbanks star Madelyn Cline was getting a facial – but it was unlike ...
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