LEXINGTON, Mass. and AMSTERDAM, the Netherlands - uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced that the U.S. Food and Drug…
A team of researchers from the University of Alberta have created a possible treatment for facioscapulohumeral muscular dystrophy (FSHD). Their work has been published in the Proceedings of the National Academy…
uniQure has recently began their Phase I/II clinical trial of AMT-130, a gene therapy for the treatment of Huntington's disease. Two patients are included so far, with one being given…
The events of the past week-- and the past few hundred years-- have brought up a lot of sadness, anger, and reckoning in our country. There are so many ways…
Recently, scientists from Emory University set out to discover whether there was any pathological link between spinocerebellar ataxia type 36 (SCA36) and amyotrophic lateral sclerosis and frontotemporal dementia (ALS/FTD). Both…
by Jodee Redmond from In the Cloud Copy Emma Larson’s parents were not particularly concerned when their daughter wasn’t standing or walking by her first birthday. They figured that plenty of children had not reached…
The month of May is Huntington's Disease Awareness Month, in which a special effort is made to improve awareness about this devastating rare disease. This year, the Huntington's Disease Society…
Ionis Pharmaceuticals, in a partnership with Roche, has announced that the enrollment has been completed for the third phase of GENERATION HD1, a trial intended to study the safety and…
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