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July 9, 2020July 9, 2020

National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US

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The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease ... Read more

July 7, 2020July 7, 2020

AFFiRiS’ New Antibody May Help Halt HD Progression, Early Data Suggests

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AFFiRiS’ C6-17, a candidate monoclonal antibody for the treatment of Huntington’s disease, is able to target and halt the spread of mutant huntingtin protein — the underlying cause of the neurodegenerative ... Read more

June 30, 2020June 30, 2020

AMT-130, uniQure’s Gene Therapy, Given to First Patient Group in Trial

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A Phase 1/2 clinical trial evaluating AMT-130 as a potential gene therapy for Huntington’s disease has “treated” its first two patients, uniQure announced. One person was given AMT-130 and the other a sham surgery ... Read more

June 23, 2020June 23, 2020

German Researcher Promotes Non-drug Alternatives for Treating Huntington’s

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At least three major pharmaceutical companies are looking into potential gene therapies for Huntington’s disease — a rare inherited condition without a cure or a treatment to stop its ... Read more

June 22, 2020June 22, 2020

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

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The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will ... Read more

June 15, 2020June 16, 2020

Austedo to Manage Chorea Safe at Higher Doses, Study Finds

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Austedo (deutetrabenazine) can be safely taken at higher than the maximum dose now approved to control involuntary movement in people with Huntington’s disease, an analysis of clinical trial data ... Read more

June 10, 2020June 10, 2020

Coalition Will Address Racial Disparities in Rare Disease Communities

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The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis ... Read more

June 9, 2020June 9, 2020

‘Hero’ Proteins Protect Other Proteins from Stress, Research Finds

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A novel class of proteins, dubbed heat-resistant obscure (Hero) proteins, can protect other proteins from a variety of cellular stresses, and also can prevent the formation of protein aggregates ... Read more

June 2, 2020June 2, 2020

Pepinemab Clinical Trial Ongoing; Top-line Results Coming Later this Year

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Vaccinex’s Phase 2 SIGNAL study, evaluating the investigational antibody pepinemab (VX15/2503) in Huntington’s disease patients with late prodromal and early disease, is ongoing and has tested pepinemab in all 265 ... Read more

May 28, 2020May 28, 2020

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

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While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene ... Read more

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