Researchers in Canada have just released positive results from their heterozygous familial hypercholesterolemia (HeFH) clinical trial. This investigation was examining the effect of Repatha on teenagers with the condition. The trial…
A new genetic test for keratoconus as well as other types of corneal dystrophy was released late last year. The test was created by Avellino and can identify biomarkers of disease. It also looks…
Rocket Pharmaceuticals has recently dosed the first patient in their trial of RP-A501, a treatment for Danon disease. According to BioSpace, this trial will test the safety, tolerability, and efficacy…
Recently, biopharmaceutical company Albireo Pharma announced positive results from a Phase 3 clinical trial. The trial, PEDFIC 1, studied the safety, efficacy, and tolerability of odevixibat for patients with progressive…
Research was presented at The Digital International Liver Congress that proved nidufexor is a safe and viable treatment for nonalcoholic steatohepatitis (NASH), according to Healio. It was shown to reduce body…
According to Parkinson's News Today, Novant Health will soon become the first provider in the Carolinas to offer MR-guided focused ultrasound (MRgFUS). The therapy, with a surgery-like technique, has no…
The CureGRIN Foundation has recently announced that its annual conference will be held virtually this year as a result of the ongoing coronavirus/COVID-19 pandemic. This year's event will be held…
According to Charcot-Marie-Tooth News, the EveryLife Foundation has began a scholarship fund to help rare disease patients in the United States. Living with a rare disease often brings challenges, many…
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