HD Focus

Today we are very happy in the Huntington community.  The announcement from Uniqure that the company has received the Regenerative Medicine Advanced Therapy (RMAT) “Designation for Investigational Gene Therapy AMT-130 […]

Vi har fått avbokningar till årets familjeläger i Hjo den 24-27 juni. Det finns alltså platser kvar och vi hoppas att just du och din familj vill komma! Lägret är nu öppet för alla att anmäla sig till. Det enda krav vi har är att alla är självgående och inte kräver extra omsorg eller medicinsk…

Inlägget Det finns platser kvar på årets familjeläger 24-27 juni i Hjo publicerades först på RHS Riksförbundet Huntingtons Sjukdom.

La Importancia de las elecciones europeas para los afectados del Huntington Las elecciones europeas son una Gran Oportunidad para darle voz a todas las familias y afectados de la enfermedad […]

La entrada Las elecciones europeas están a la vuelta de la esquina. Descubre por qué las familias afectadas por la EH deberíamos ir a votar se publicó primero en .

 9 июня в 12.00 часов состоится Информационный (поликлинический) день для семей с болезнью Гентингтона .На инфо дне вы получите бесплатные консультации у лучших специалистов в области изучения и лечения болезни… More »

The post Информационный (поликлинический) день 9 июня 2024 г. appeared first on Центр помощи пациентам с орфанными заболеваниями.

The Postcards are a series of short videos aimed at non-scientists that convey the major themes, scientific breakthroughs, and clinical trial updates from each year’s Huntington’s Disease Therapeutics Conference held (most usually) in Palm Springs, California. Beginning in 2006, this series of conferences is a unique forum where researchers from academia meet with pharmaceutical and […]

Hamburg, Germany, 28 May 2024: Evotec SE (Frankfurt Stock Exchange: EVT, MDAX/TecDAX, ISIN: DE0005664809; NASDAQ: EVO) has announced the extension of its 20-year collaboration with CHDI Foundation, Inc. (“CHDI”). CHDI is a privately funded nonprofit biomedical research organization exclusively dedicated to collaboratively developing therapeutics that will substantially improve the lives of those affected by Huntington’s […]

Huntingtin (HTT)-lowering and somatic expansion have been two of the hottest topics in Huntington’s disease (HD) research in the past decade. Recent work from a team at Massachusetts General Hospital detailed a serendipitous overlap between the two – certain HTT-lowering drugs can also help regulate the ongoing CAG repeat expansion. Seemingly, this could allow researchers to kill two birds with one stone using a single drug. But there’s more to this story.

CAG expansion causes toxicity

The CAG repeat within the HTT gene is the nefarious player leading to HD. This repeat can expand in some cells over time, which is the biological phenomenon known as somatic expansion. We’ve talked a lot about somatic expansion lately, which you can read more about in this recent article.

El pasado jueves, 11 de abril de 2024 tuvimos la oportunidad de asistir al XVIII Congreso Internacional de Enfermedades Neurodegenerativas de CIBERNED que se llevó a cabo en Bilbao, en […]

La entrada Avances de investigación en la Enfermedad de Huntington se publicó primero en .

日時：2024年7月6日（土）　13：30～17：00　ハイブリッド形式場所：東京駅周辺の会議室 　　　お問合せは事務局メール　jhdn2000@jhdn.org まで プログラム（仮）　13：30　総会14：30　講演 […]