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
Prilenia, kliinisen vaiheen biotekniikkayhtiö, joka on keskittynyt kiireelliseen tehtävään kehittää uusia terapeuttisia lääkkeitä hermoston rappeutumissairauksien ja neurologisten kehityshäiriöiden etenemisen hidastamiseksi, on jättänyt Euroopan lääkevirastolle (EMA) eurooppalaisen myyntilupahakemuksen (MAA) pridopidiinille Huntingtonin taudin hoitona. Myyntilupahakemus
Kuulun Huntingtonin tautia sairastavan suvun kolmanteen sukupolveen, mutta vasta vuonna 2024 toisen isäni F. Cookin väsymättömien ponnistelujen ansiosta saimme varmistettua, miten tauti on levinnyt suvussa. Äidin isoisoisäni, ranskalainen 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,
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
Prilenia, kliinisen vaiheen biotekniikkayhtiö, joka on keskittynyt kiireelliseen tehtävään kehittää uusia terapeuttisia lääkkeitä hermoston rappeutumissairauksien ja neurologisten kehityshäiriöiden etenemisen hidastamiseksi, on jättänyt Euroopan lääkevirastolle (EMA) eurooppalaisen myyntilupahakemuksen (MAA) pridopidiinille Huntingtonin taudin hoitona. Myyntilupahakemus
Kuulun Huntingtonin tautia sairastavan suvun kolmanteen sukupolveen, mutta vasta vuonna 2024 toisen isäni F. Cookin väsymättömien ponnistelujen ansiosta saimme varmistettua, miten tauti on levinnyt suvussa. Äidin isoisoisäni, ranskalainen 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,
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