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 for Huntington, Dimitri visited several HD associations and families, trying to raise awareness for Huntington’s. “The Present is directed by
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 tiered double-digit royalties on ex-U.S. net sales –– Novartis will assume global development, manufacturing and commercial responsibilities following completion of placebo-controlled portion of
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 further development of dalzanemdor CAMBRIDGE, Mass.–(BUSINESS WIRE)–Nov. 20, 2024– Sage Therapeutics, Inc. (Nasdaq: SAGE) today announced topline results from the Phase 2
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 Disease Community, by Christy Dearien, with a foreword by Jimmy Pollard. In Livable Lives, Christy shares her own family’s HD
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 type 1 and similar triplet repeat expansion diseases, today announces the successful close of its £35 million (c.$43 million) Series
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 eligible for priority review and accelerated approval. This follows the release of 12 month interim data from PTC’s Phase 2 study, which
Prilenia, a clinical stage biotechnology company focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders, has filed a European Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) for pridopidine as a treatment for Huntington’s disease (HD). An MAA
I belong to the third generation of a family affected by Huntington’s Disease, but it wasn’t until 2024, thanks to the tireless efforts of my second father, F. Cook, that we were able to confirm how the disease entered the family. My maternal great-grandfather, a Frenchman named Charles Novel-Catin (1883-1936),
Abu Dhabi, UAE; June 19, 2024: IROS, an Abu Dhabi-based contract research organization (part of the M42 group), has partnered with Prilenia Therapeutics, a clinical-stage biotech company, and the International Huntington’s Disease Association (IHA) to plan the first-ever clinical trial for Huntington’s disease (HD) in the UAE and MENA region,
“We are very pleased with these new data demonstrating a statistically significant, dose-dependent slowing of the progression of Huntington’s disease and lowering of NfL in the CSF at 24 months,” stated Walid Abi-Saab, M.D., chief medical officer of uniQure. “We believe this is the first clinical trial of any investigational medicine
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 for Huntington, Dimitri visited several HD associations and families, trying to raise awareness for Huntington’s. “The Present is directed by
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 tiered double-digit royalties on ex-U.S. net sales –– Novartis will assume global development, manufacturing and commercial responsibilities following completion of placebo-controlled portion of
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 further development of dalzanemdor CAMBRIDGE, Mass.–(BUSINESS WIRE)–Nov. 20, 2024– Sage Therapeutics, Inc. (Nasdaq: SAGE) today announced topline results from the Phase 2
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 Disease Community, by Christy Dearien, with a foreword by Jimmy Pollard. In Livable Lives, Christy shares her own family’s HD
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 type 1 and similar triplet repeat expansion diseases, today announces the successful close of its £35 million (c.$43 million) Series
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 eligible for priority review and accelerated approval. This follows the release of 12 month interim data from PTC’s Phase 2 study, which
Prilenia, a clinical stage biotechnology company focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders, has filed a European Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) for pridopidine as a treatment for Huntington’s disease (HD). An MAA
I belong to the third generation of a family affected by Huntington’s Disease, but it wasn’t until 2024, thanks to the tireless efforts of my second father, F. Cook, that we were able to confirm how the disease entered the family. My maternal great-grandfather, a Frenchman named Charles Novel-Catin (1883-1936),
Abu Dhabi, UAE; June 19, 2024: IROS, an Abu Dhabi-based contract research organization (part of the M42 group), has partnered with Prilenia Therapeutics, a clinical-stage biotech company, and the International Huntington’s Disease Association (IHA) to plan the first-ever clinical trial for Huntington’s disease (HD) in the UAE and MENA region,
“We are very pleased with these new data demonstrating a statistically significant, dose-dependent slowing of the progression of Huntington’s disease and lowering of NfL in the CSF at 24 months,” stated Walid Abi-Saab, M.D., chief medical officer of uniQure. “We believe this is the first clinical trial of any investigational medicine
English 
Arabic 
Bulgarian 
Chinese 
Czech 
Danish 
Dutch 
Estonian 
Finnish 
French 
German 
Greek 
Hungarian 
Indonesian 
Italian 
Japanese 
Latvian 
Lithuanian 
Polish 
Portuguese 
Romanian 
Russian 
Slovak 
Slovenian 
Spanish 
Swedish 
Turkish 
Ukrainian