What a gene therapy win means for the Huntington’s disease pipeline

“Beyond UniQure, the pipeline includes highly promising approaches that will ideally move the goal from merely managing ...
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Experimental gene therapy found to slow Huntington's disease progression, company says

An experimental gene therapy was found to slow the progression of Huntington's disease in a "pivotal" Phase 1/2 study, according to drugmaker uniQure, marking a major step toward a potential first ...

For the first time, Huntington’s disease slowed: what scientists achieved and how gene therapy blocks toxic brain protein

Huntington's disease has been successfully treated for the first time with an experimental gene therapy called AMT-130, which uses a clever genetic technique to stop the toxic protein that causes the ...

Novartis to pursue SMA drug branaplam in Huntington's disease

Novartis is seeking to repurpose its investigational oral spinal muscular atrophy (SMA) drug branaplam to treat Huntington's disease, the Swiss drugmaker said on Wednesday, as it plans a clinical ...
Futurism

In Major Breakthrough, Scientists Successfully Treat Devastating Brain Disease Huntington’s for the First Time

A research team says that for the first time in history, it’s successfully treated Huntington’s disease, a devastating and inherited disorder that kills nerve cells and leads to rapid decline and ...
Business Wire

Prilenia and Ferrer Provide Update on European Regulatory Process for Pridopidine in Huntington’s Disease

Pridopidine (45 mg twice daily) is a potent and selective, orally administered sigma-1 receptor (S1R) agonist which stimulates key neuroprotective mechanisms often impaired in neurodegenerative ...
WRAL

Experimental gene therapy found to slow Huntington’s disease progression, company says

“For the approximate 42,000 Americans and their families living with Huntington’s disease, this is a truly transformative development,” Gray, who was not involved in the research, said in an email.
CNN

Experimental gene therapy found to slow Huntington’s disease progression, company says

An experimental gene therapy was found to slow the progression of Huntington’s disease in a “pivotal” Phase 1/2 study, according to drugmaker uniQure, marking a major step toward a potential first ...