Huntington’s Disease News

June 15, 2021June 15, 2021

Get Ready for NORD’s Patient and Family Forum, Set for June 26-27

Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the ... Read more

June 15, 2021June 15, 2021

Galyan Bio Plans to Start Clinical Testing of Oral Therapy in 2022

Huntington's Disease News

Galyan Bio plans to initiate a clinical trial of a small molecules candidate that aims to slow the progression of Huntington’s disease next year, working in with Bicoll GmbH. Galyan Bio is developing and optimizing ... Read more

June 8, 2021June 8, 2021

Rare 2030 Action Campaign Pushing for European Plan for Rare Diseases

Huntington's Disease News

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in ... Read more

June 8, 2021June 8, 2021

Levels of KP Metabolites in Blood, CSF Unlikely as Disease Biomarkers

Huntington's Disease News

Metabolites seen as potential biomarkers of Huntington’s disease — called kynurenine pathway metabolites — showed little evidence of Huntington’s-specific activity in a recent study. While researchers could not entirely rule out ... Read more

June 4, 2021June 4, 2021

Advocates Can Try for Grants Ahead of Rare Disease Week on Capitol Hill

Huntington's Disease News

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in ... Read more

June 1, 2021June 1, 2021

Board OKs AMT-130 Gene Therapy Trial to Enroll for High-dose Group

Huntington's Disease News

A Phase 1/2 trial testing the safety and efficacy of AMT-130, uniQure’s experimental gene therapy for Huntington’s disease, can continue dosing after a positive recommendation from an independent board. The data safety ... Read more

May 25, 2021May 25, 2021

PROOF-HD Phase 3 Pridopidine Trial Reaches 25% Enrollment

Huntington's Disease News

Prilenia Therapeutics has enrolled more than 120 patients in PROOF-HD, a Phase 3 clinical trial investigating the efficacy and safety of oral pridopidine in early stage Huntington’s disease. That amounts ... Read more

May 18, 2021May 18, 2021

1st Group Enrolled in Trial of uniQure’s AMT-130 Gene Therapy

Huntington's Disease News

uniQure has completed patient enrollment for the first group participating in a Phase 1/2 trial assessing the safety and efficacy of AMT-130, its experimental gene therapy for Huntington’s disease. “This is ... Read more

May 11, 2021May 11, 2021

Nonprofit Pushes for US Bill to End Wait for Patient Benefits

Huntington's Disease News

U.S. lawmakers have again introduced legislation that would speed up the process for Huntington’s disease (HD) patients to get Medicare and Social Security Disability Insurance (SSDI) benefits. The Huntington’s Disease ... Read more

May 7, 2021May 7, 2021

Decision to Stop Dosing in Tominersen Trial ‘Pretty Hard to Take’

Huntington's Disease News

Sarah Lynn Mays learned she wouldn’t be returning to Northwestern University for her scheduled intrathecal injection of tominersen when her younger sister called to give the news — Roche was stopping ... Read more

May 6, 2021May 6, 2021

GENERATION HD1 Trial Fails to Show Benefits of Tominersen

Huntington's Disease News

Treatment with tominersen failed to show evidence of clinical benefit in adults with Huntington’s disease during the Phase 3 clinical trial GENERATION HD1. Roche, the company developing tominersen, decided to pause ... Read more

May 4, 2021May 4, 2021

‘Let’s Talk About’ Huntington’s Disease Awareness in May

Huntington's Disease News

Activities are underway worldwide to mark Huntington’s Disease Awareness Month, set aside each year to call attention to the neurodegenerative disorder that is thought to affect three to seven per ... Read more

April 27, 2021April 27, 2021

FDA Gives Go-ahead for Phase 1/2 Trial Testing VY-HTT01 Gene Therapy

Huntington's Disease News

The U.S. Food and Drug Administration (FDA) has granted Voyager Therapeutics permission to start a clinical trial testing VY-HTT01, its investigational gene therapy for Huntington’s disease. Voyager is planning to launch ... Read more

April 21, 2021April 21, 2021

Quick Approval Given COVID Vaccines Raise Concerns, Rare Disease Patients Say

Huntington's Disease News

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine ... Read more

April 20, 2021April 20, 2021

Protein With ‘Switch’ Sensitive to Clumping May Aid in Treating Disease

Huntington's Disease News

A chaperone protein called DnaJB8 has an internal molecular “switch” that likely regulates how cells dispose of protein aggregates, like those that cause Huntington’s disease. These findings were detailed in the study ... Read more