Category: News

RDI Launches Youth leadership programme

Rare Diseases International (RDI), announced the launch of the RDI Youth Leadership Programme, an initiative designed to engage and empower young people within the rare disease community. This programme aims […]

Twelve-year-old publishes children’s book about Huntington’s Disease

Toronto student writes book so others will treat her grandpa kindly December 11, 2024 — Toronto, Ontario, Canada Most people have never heard of Huntington’s disease. This lack of awareness […]

UniQure announces alignment with FDA on key elements of accelerated approval pathway for AMT-130

Yesterday, uniQure issued a press release announcing alignment with the US Food and Drug Administration (FDA) on key elements of an accelerated approval pathway for uniQure‘s AMT-130 program in Huntington’s […]

“The Present”, documentary featuring Dimitri Poffé, is now available on Youtube

Dimitri Poffé is an already known member of the HD Community. After testing positive for Huntington’s Disease, Dimitri embraced a bicycle journey through South America. In this project, called Explore […]

PTC Therapeutics Enters into a Global License and Collaboration Agreement with Novartis for PTC518 Huntington’s Disease Program

December 2, 2024 – PTC to receive $1.0B in cash at closing –– PTC is eligible to receive up to $1.9B in development, regulatory and sales milestones –– PTC to share profits in the U.S. and […]

Sage Therapeutics ceases development of dalzanemdor in HD

The Phase 2 DIMENSION Study did not meet its primary endpoint Dalzanemdor was generally well-tolerated; no new safety signals were observed Based on these data, the Company does not plan […]

The Power of Partnership. Switzerland and China set an Example.

In this video, Professor Jean-Marc Burgunder, head of the Huntington’s Disease (HD) centre in Bern Switzerland, and Xi Cao, President of the Chinese HD Association talk about the benefits of […]

Livable Lives – a new book that highlights stories from people impacted by HD around the world

PRESS RELEASE – October 25, 2024, Moscow, Idaho, USAWe are pleased to announce the launch of a new book about Huntington’s disease (HD) called, Livable Lives: Conversations with the Huntington’s […]

LoQus23 Therapeutics announces £35 million financing to advance a new drug aimed at inhibiting somatic expansion in Huntington’s Disease

Cambridge, UK, 2 October 2024 – LoQus23 Therapeutics Ltd (“LoQus23”), a private biotechnology company investigating small molecule drugs that could stop DNA instability and slow neurodegeneration in Huntington’s Disease, myotonic dystrophy […]

FDA Grants Fast Track Designation to PTC518 Huntington’s Disease Program

PTC518 is an oral medication that reduces the production of the mutated Huntingtin protein that causes disease progression.   Programs with Fast Track designation can benefit from early interactions with the FDA, and may be […]