Rare Disease Report

Huntingtons 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.
The study of IONIS-HTTRx was completed. The Phase 1/2a evaluated the first therapy in clinical development intended to target the underlying cause of Huntington’s Disease (HD).
RDR spoke with George Yohrling of the HDSA about AMT-130, uniQure N.V.’s gene therapy which received an Orphan Drug Designation in October.
With altering the immune system in HD patients emerging as such a hot topic, Rare Disease Report sat down with George Yohrling to get his thoughts on whether this method is worth pursuing.
A new study revealed that ATF5 proteins can become implanted in other substances in the brains of patients with Huntington’s disease.
Is the immune system involved in the progression of HD? And can drugs that target the immune system help HD patients?
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