Gain Therapeutics' lead drug candidate, GT-02287, is in clinical development for the treatment of Parkinson's disease (PD) with or without a GBA1 mutation. The orally administered, brain-penetrant ...

Alexion is no stranger to dealing with NICE's rare disease drug review system – its other drug Soliris was the first to be appraised by NICE, and gained recommendation in December 2014 after a ...

Belite Bio, Inc., a clinical-stage biopharmaceutical company focusing on treatments for degenerative retinal diseases, announced that it will present two posters at the upcoming ARVO Annual ...

The NHS is “not working” for people in the UK with rare diseases as they are left to “fall through the cracks,” a damning new report has claimed. Nearly three in ten (30 per cent ...

There is currently no cure. "Historical data is quite limited because it is a rare disease, but 90% of kids, they say, will pass before the age of four. We are hopeful that will not be the case ...

For the past 20 years, Janet Edmunson has run support groups for patients with rare neurological diseases that have no known treatment or cure. What they want to know, Edmunson said, is “‘When ...

Dr Mickey Mehta has issued a warning on his social media page, advising against eating bread on a regular basis due to the risk of developing a rare disease known as Auto Brewery Syndrome. Dr Mehta ...

This was tragically echoed in the story of the American teen who developed the disease. Her case is reminiscent of the 2019 Evali crisis (e-cigarette or vaping product use-associated lung injury ...

Of the over 7,000 rare diseases currently identified, 95% do not have an FDA-approved treatment or cure. This is where I come in. Dana-Farber Cancer Institute in Boston, where I am treated ...

at the upcoming Digestive Disease Week Conference (DDW), to be held May 3-6, 2025, in San Diego. IBSRELA is approved by the U.S. Food and Drug Administration to treat irritable bowel syndrome with ...