Tag: 上海后花园KZW

Amicus therapy shows durable muscle function improvements in Pompe disease patients

first_img About the Author Reprints John Crowley and his daughter, Megan, whose illness inspired him to look for a treatment. Crowley family @adamfeuerstein GET STARTED Amicus Therapeutics (FOLD) is presenting updated results Wednesday from a small clinical trial involving patients with the rare and debilitating Pompe disease. Amicus’ drug continues to improve walking performance and stabilize lung function in patients followed for up to one year.Data from this same Pompe disease study were last presented by Amicus in October. Daily reporting and analysis The most comprehensive industry coverage from a powerhouse team of reporters Subscriber-only newsletters Daily newsletters to brief you on the most important industry news of the day STAT+ Conversations Weekly opportunities to engage with our reporters and leading industry experts in live video conversations Exclusive industry events Premium access to subscriber-only networking events around the country The best reporters in the industry The most trusted and well-connected newsroom in the health care industry And much more Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr. Log In | Learn More What is it? STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond. What’s included?center_img By Adam Feuerstein Feb. 7, 2018 Reprints [email protected] Adam Feuerstein Senior Writer, Biotech Adam is STAT’s national biotech columnist, reporting on the intersection of biotech and Wall Street. He’s also a co-host of “The Readout LOUD” podcast. Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+. First 30 days free. GET STARTED Amicus therapy shows durable muscle function improvements in Pompe disease patients Biotech Tags biotechdrug developmentrare diseaseSTAT+last_img read more