Startup Glycomine Lands $115M for Clinical Trial in Ultra-Rare Disease With No Approved Drugs

Glycomine’s lead program is an experimental treatment for PMM2-CDG, an ultra-rare enzyme deficiency often misdiagnosed as cerebral palsy. With encouraging results from an open-label Phase 2 study, the biotech startup is now preparing for a placebo-controlled test that could potentially support a regulatory submission. The post Startup Glycomine Lands $115M for Clinical Trial in Ultra-Rare…

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Medicare’s Hidden Information Hurts People & Policy

By MICHAEL MILLENSON Open enrollment season for Medicare, which began Oct. 15 and ends Dec. 7, triggers a deluge of information about various options. Since I’m a health care consultant and researcher as well as a Medicare beneficiary, I’ve looked critically at what we’re told and what we’re not. Unfortunately, information crucial both for the…

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Liver Failure-Associated Death Reported in Patient Treated With Sarepta Gene Therapy Elevidys

Sarepta Therapeutics said the Duchenne muscular dystrophy patient recently had a cytomegalovirus infection, which can damage the liver and may have contributed to the fatality. It’s the first patient death out of more than 800 patients treated with Elevidys, a gene therapy for the rare muscle-wasting disease. The post Liver Failure-Associated Death Reported in Patient…

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