Rare Disease Report

Huntingtons Disease

Teva Pharmaceuticals, the HDSA, and the ALS Association are partnering for the Teva CNS Target Identification Challenge, a crowdsourcing effort to seek novel targets with therapeutic potential.
It was recently revealed that a newly-discovered biochemical pathway could assist scientists develop new ways to protect cells against the oxidative stress that is commonly associated with Huntington’s disease.
Ionis Pharmaceuticals reported positive top-line data from a completed Phase 1/2 study of RG6042 (Ionis HTTRx) in people with early stage Huntington’s disease .
George Yohrling, Ph.D., Senior Director of Mission and Science at the HDSA discusses the challenges that come with working with patients with HD, and communicating with their family members and caregivers.
Yesterday, nonprofit organization BioPontis Alliance and Massachusetts General Hospital, the largest hospital-based research program in the United States, announced an agreement to target new treatments for Huntington’s disease.
Rockefeller University scientists have observed the effects of Huntington’s disease in neurons as early as conception, and suggest therapies that block HTT protein activity may be doing more harm than good.
New data concludes that neurofeedback training could potentially increase brain connectivity in patients with Huntington’s disease (HD).
uniQure was granted Orphan Medicinal Product Designation from the European Medicines Agency for AMT-130, an investigational gene therapy for the treatment of Huntington’s disease.
In 2018, uniQure N.V. expects to advance the clinical development of AMT-130, its investigational gene therapy for the treatment of Huntington’s disease.
New data published in the Orphanet Journal of Rare Diseases has concluded that MiR-9* in peripheral leukocyte could potentially serve as a signature feature of neurodegeneration in Huntington’s disease patients.
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