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 Ler mais...
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 Ler mais...
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 Ler mais...
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 Ler mais...
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 Ler mais...
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 Ler mais...
A Prilenia, uma empresa de biotecnologia em fase clínica centrada na missão urgente de desenvolver novas terapêuticas para retardar a progressão de doenças neurodegenerativas e perturbações do desenvolvimento neurológico, apresentou um pedido de autorização de introdução no mercado europeu (MAA) à Agência Europeia de Medicamentos (EMA) para a pridopidina como tratamento para a doença de Huntington (DH). Um MAA Ler mais...
Abu Dhabi, EAU; 19 de junho de 2024: A IROS, uma organização de investigação contratada sediada em Abu Dhabi (parte do grupo M42), estabeleceu uma parceria com a Prilenia Therapeutics, uma empresa de biotecnologia em fase clínica, e a Associação Internacional da Doença de Huntington (IHA) para planear o primeiro ensaio clínico para a doença de Huntington (DH) nos EAU e na região MENA, Ler mais...
"Estamos muito satisfeitos com estes novos dados, que demonstram um abrandamento estatisticamente significativo e dependente da dose da progressão da doença de Huntington e uma redução da NfL no LCR aos 24 meses", afirmou Walid Abi-Saab, M.D., diretor médico da uniQure. "Acreditamos que este é o primeiro ensaio clínico de qualquer medicamento experimental Ler mais...
Hoje, a Wave Life Sciences anunciou resultados positivos do SELECT-HD, o nosso ensaio de Fase 1b/2a controlado por placebo que avalia a terapia experimental WVE-003. Estes resultados demonstram que o WVE-003 reduz seletivamente a proteína huntingtina mutante tóxica (mHTT) e preserva a proteína huntingtina saudável e de tipo selvagem (wtHTT) em indivíduos com doença de Huntington. Resultados do ensaioNa análise, foi comparado Ler mais...
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