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 är ett oralt läkemedel som minskar produktionen av det muterade Huntingtin-proteinet som orsakar sjukdomsutveckling. Program med Fast Track-beteckning kan dra nytta av tidiga interaktioner med FDA och kan vara berättigade till prioriterad granskning och påskyndat godkännande. Detta följer på publiceringen av 12 månaders interimsdata från PTC:s fas 2-studie, som
Prilenia, ett bioteknikbolag i klinisk fas med fokus på att utveckla nya läkemedel för att bromsa utvecklingen av neurodegenerativa sjukdomar och neurologiska utvecklingsstörningar, har lämnat in en europeisk ansökan om marknadsgodkännande (MAA) till den europeiska läkemedelsmyndigheten (EMA) för pridopidin som behandling av Huntingtons sjukdom (HS). En MAA
Jag tillhör den tredje generationen i en familj som drabbats av Huntingtons sjukdom, men det var inte förrän 2024, tack vare min andre far F. Cooks outtröttliga ansträngningar, som vi kunde bekräfta hur sjukdomen kom in i familjen. Min mormors farfars far, en fransman vid namn Charles Novel-Catin (1883-1936),
Abu Dhabi, Förenade Arabemiraten; 19 juni 2024: IROS, en Abu Dhabi-baserad kontraktsforskningsorganisation (del av M42-gruppen), har ingått partnerskap med Prilenia Therapeutics, ett bioteknikföretag i klinisk fas, och International Huntington's Disease Association (IHA) för att planera den första kliniska prövningen någonsin för Huntingtons sjukdom (HD) i Förenade Arabemiraten och MENA-regionen,
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 är ett oralt läkemedel som minskar produktionen av det muterade Huntingtin-proteinet som orsakar sjukdomsutveckling. Program med Fast Track-beteckning kan dra nytta av tidiga interaktioner med FDA och kan vara berättigade till prioriterad granskning och påskyndat godkännande. Detta följer på publiceringen av 12 månaders interimsdata från PTC:s fas 2-studie, som
Prilenia, ett bioteknikbolag i klinisk fas med fokus på att utveckla nya läkemedel för att bromsa utvecklingen av neurodegenerativa sjukdomar och neurologiska utvecklingsstörningar, har lämnat in en europeisk ansökan om marknadsgodkännande (MAA) till den europeiska läkemedelsmyndigheten (EMA) för pridopidin som behandling av Huntingtons sjukdom (HS). En MAA
Jag tillhör den tredje generationen i en familj som drabbats av Huntingtons sjukdom, men det var inte förrän 2024, tack vare min andre far F. Cooks outtröttliga ansträngningar, som vi kunde bekräfta hur sjukdomen kom in i familjen. Min mormors farfars far, en fransman vid namn Charles Novel-Catin (1883-1936),
Abu Dhabi, Förenade Arabemiraten; 19 juni 2024: IROS, en Abu Dhabi-baserad kontraktsforskningsorganisation (del av M42-gruppen), har ingått partnerskap med Prilenia Therapeutics, ett bioteknikföretag i klinisk fas, och International Huntington's Disease Association (IHA) för att planera den första kliniska prövningen någonsin för Huntingtons sjukdom (HD) i Förenade Arabemiraten och MENA-regionen,
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