Huntington’s Disease News

June 14, 2022June 14, 2022

New Human Cell Model From Bit.Bio Aims to Advance Research, Therapies

Bit.Bio has launched a new human cell model to advance research and accelerate therapeutic development for Huntington’s disease. The cells, called ioGlutamatergic Neurons HTT-50CAG/T, are engineered to carry a specific mutation ... Read more

June 7, 2022June 7, 2022

NORD’s ‘Living Rare, Living Stronger’ Patient, Family Forum Set for June 26

Huntington's Disease News

The National Organization for Rare Disorders’ (NORD) “Living Rare, Living Stronger Patient and Family Forum” is back in person on June 26 for a day of learning and networking in ... Read more

May 24, 2022May 24, 2022

Pathway Activation Clears Signs of Huntington’s in Mouse Model: Study

Huntington's Disease News

Activating the JAK2-STAT3 pathway in astrocyte cells reduced multiple hallmark features of Huntington’s disease — including the toxic clumping of the huntingtin protein in nerve cells — from the brains ... Read more

May 17, 2022May 17, 2022

FDA Names Valbenazine an Orphan Drug for Chorea With Huntington’s

Huntington's Disease News

The U.S. Food and Drug Administration (FDA) has designated valbenazine an orphan drug as a potential treatment to control the involuntary, jerky movements known as chorea in people with Huntington’s ... Read more

May 10, 2022May 10, 2022

Huntington’s Protein Clumps Form Distinct Structures in Nerve Cells

Huntington's Disease News

Clumps of protein resulting from HTT mutations, the underlying cause of Huntington’s disease, form distinct structures in different parts of the cell, according to detailed imaging analysis. The findings suggest ... Read more

May 3, 2022May 3, 2022

HDSA Launches Social Media Effort for Huntington’s Awareness Month

Huntington's Disease News

Some 41,000 U.S. residents are thought to live with Huntington’s disease (HD), a rare progressive disorder that breaks down brain nerve cells. During May, and as part of Huntington’s Disease ... Read more

April 29, 2022April 29, 2022

Organizations Rally to Help Ukrainian Rare Disease Patients

Huntington's Disease News

A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ... Read more

April 27, 2022April 27, 2022

#AAN2022 – Pepinemab Appears to Help Patients With Cognitive Issues

Huntington's Disease News

The experimental therapy pepinemab can aid cognition in people with early Huntington’s disease who showed signs of cognitive impairment when they started treatment, new analyses from the Phase 2 SIGNAL clinical ... Read more

April 19, 2022April 19, 2022

Europe Awaits Proposed New Framework for Sharing Health Data

Huntington's Disease News

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health ... Read more

April 14, 2022April 14, 2022

Telehealth Added to Latest Digital-friendly NORD State Report Card

Huntington's Disease News

The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues ... Read more

April 12, 2022April 12, 2022

#AAN2022 – Valbenazine Quickly Eases Chorea in KINECT-HD Trial

Huntington's Disease News

Treatment with oral valbenazine, a dopamine-modulating medicine, significantly eased the jerky, involuntary movements of chorea after as little as two weeks for Huntington’s disease patients in a completed Phase 3 clinical trial. ... Read more

April 5, 2022April 5, 2022

Phase 2 Trial of Oral Therapy PTC518 Launched in US

Huntington's Disease News

A Phase 2 trial evaluating PTC518, an investigational treatment for Huntington’s disease, has started in the U.S., PTC Therapeutics announced. The trial, called PIVOT-HD, will run across clinical sites worldwide ... Read more

March 29, 2022March 29, 2022

Time Perception Impaired – but Only in Patients With Early Symptoms

Huntington's Disease News

The perception of time is impaired in people with symptomatic Huntington’s disease in its early stages — but not in patients without symptoms, a study revealed. These findings suggest that ... Read more

March 25, 2022March 25, 2022

Increased Education Crucial to Improving Rare Disease Care, Survey Finds

Huntington's Disease News

Healthcare providers involved in diagnosing and treating rare diseases believe that increased physician education and collaboration with specialized facilities will have the greatest positive impact on treating these conditions over ... Read more

March 22, 2022March 22, 2022

First 2 Groups Enrolled in Phase 1/2 Trial of Gene Therapy AMT-130

Huntington's Disease News

The first two dosing groups in a Phase 1/2 trial of AMT-130, uniQure’s investigational gene therapy for Huntington’s disease, are now fully enrolled. The trial (NCT04120493), being conducted across 12 ... Read more