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March 10, 2020March 10, 2020

UniQure Presents More Data Supporting the Effectiveness of Gene Therapy Candidate AMT-130

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New preclinical data continues to support uniQure’s AMT-130 investigational gene therapy for Huntington’s disease. Results reveal the therapy lowered the levels of toxic huntingtin protein in nerve cells derived from ... Read more

March 5, 2020March 5, 2020

RG6042, Potential DMT in Phase 3 Trial, Named Orphan Drug in Japan

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A potential treatment called RG6042, which target all forms of the mutant huntingtin protein (mHTT) — the underlying cause of Huntington’s disease, has been named an orphan drug by the ... Read more

March 5, 2020March 5, 2020

Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare

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In recognition of Rare Disease Day Feb. 29, BioNews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the ... Read more

March 3, 2020March 3, 2020

Antihypertensives Linked to Later Onset, Milder Disease in Huntington’s Patients, Large Study Finds

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Use of antihypertensives — medications used to treat high blood pressure — is associated with milder disease, slower progression, and delayed clinical onset in people with Huntington’s disease, a ... Read more

March 2, 2020March 2, 2020

Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29

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Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, ... Read more

February 27, 2020February 27, 2020

High Levels of Protein Linked to Parkinson’s Found in Huntington’s Patients, Study Says

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Alpha-synuclein, the protein implicated in Parkinson’s disease, may play a role in Huntington’s disease after researchers found elevated levels of the protein in patients, a recent study suggests. The ... Read more

February 26, 2020February 26, 2020

Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar

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Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those looking to begin the complex process in its Feb. ... Read more

February 25, 2020February 25, 2020

Rural Vermont Couple Talk of Huntington’s ‘Crippling’ Financial and Emotional Toll

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Linda Lacroix of Milton, Vermont, never imagined that at the age of 68, she’d be working as a lunch lady at the local high school cafeteria. But that’s what ... Read more

February 20, 2020March 3, 2020

Patients, Supporters Worldwide Recognizing Rare Disease Day 2020

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An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and ... Read more

February 20, 2020February 20, 2020

Gait Variability Reflects Huntington’s Severity, According to New Sensor-Based Study of Walking Problems

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Digital gait measurements collected through sensors can be used as a biomarker for walking impairments in people with Huntington’s disease (HD), a new study suggests. Individuals with HD were ... Read more

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