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 disease. The key takeaways are as follows: · The U.S. Food and Drug Administration (FDA) agrees that data from ongoing
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
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
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, 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,
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 disease. The key takeaways are as follows: · The U.S. Food and Drug Administration (FDA) agrees that data from ongoing
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
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
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, 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,
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