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March 24, 2020March 24, 2020

‘Our Odyssey’ Nonprofit Connects Young Adults Who Have Rare Diseases

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As a high-school student in the suburbs of Boston, Seth Rotberg watched his mother’s mental and physical health deteriorate because of Huntington’s disease. Rotberg was embarrassed to go out in ... Read more

March 20, 2020March 20, 2020

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

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Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see ... Read more

March 19, 2020March 19, 2020

Gene Therapy Reprograms Brain Cells in Mouse Model, Improves Motor Function

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In a proof-of-principle experiment, a new gene therapy approach with the potential to treat people with Huntington’s disease has been shown to effectively convert astrocyte brain cells of mice ... Read more

March 17, 2020March 17, 2020

Global Enroll-HD Study Aims for Up to 30,000 Patients by 2023

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Enroll-HD, the world’s largest observational study of Huntington’s disease, hopes to add thousands of patients to its registry, reaching a possible total of 30,000 over the next three years. Georg ... Read more

March 12, 2020March 12, 2020

Roche Suspends Phase 1 Trial Testing Tominersen (RG6042) for Huntington’s

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A Phase 1 clinical trial testing Roche’s investigational therapy tominersen for Huntington’s disease has been placed on hold after two cases of infection were found during the study. The infections ... Read more

March 10, 2020March 10, 2020

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

Huntington's Disease News, News

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still ... Read more

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

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