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 ir iekšķīgi lietojams medikaments, kas samazina mutētā hantingtīna proteīna, kurš izraisa slimības progresēšanu, veidošanos. Programmas, kurām piešķirts Fast Track apzīmējums, var gūt labumu no agrīnas mijiedarbības ar FDA, un tās var pretendēt uz prioritāru pārskatīšanu un paātrinātu apstiprināšanu. Pēc tam, kad tika publicēti PTC 2. fāzes pētījuma 12 mēnešu starpposma dati, kas liecina, ka
Prilenia, klīniskās stadijas biotehnoloģiju uzņēmums, kas koncentrējas uz steidzamu misiju izstrādāt jaunas terapijas zāles, lai palēninātu neirodeģeneratīvo slimību un neiroloģiskās attīstības traucējumu progresēšanu, ir iesniedzis Eiropas Zāļu aģentūrai (EMA) Eiropas tirdzniecības atļaujas pieteikumu (MAA) attiecībā uz pridopidīnu kā Hantingtona slimības (HD) ārstēšanas līdzekli. MAA
Es piederu jau trešajai Hantingtona slimības skartās ģimenes paaudzei, taču tikai 2024. gadā, pateicoties mana otrā tēva F. Kuka nenogurstošajām pūlēm, mums izdevās noskaidrot, kā šī slimība ienāca ģimenē. Mans vecvectēvs no mātes puses, francūzis vārdā Šarls Novels Katēns (Charles Novel-Catin, 1883-1936),
Abū Dabī, AAE; 2024. gada 19. jūnijs: IROS, Abū Dabī bāzēta līgumpētniecības organizācija (M42 grupas daļa), sadarbojas ar biotehnoloģiju uzņēmumu Prilenia Therapeutics un Starptautisko Hantingtona slimības asociāciju (IHA), lai plānotu pirmo Hantingtona slimības (HD) klīnisko pētījumu AAE un MENA reģionā,
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 ir iekšķīgi lietojams medikaments, kas samazina mutētā hantingtīna proteīna, kurš izraisa slimības progresēšanu, veidošanos. Programmas, kurām piešķirts Fast Track apzīmējums, var gūt labumu no agrīnas mijiedarbības ar FDA, un tās var pretendēt uz prioritāru pārskatīšanu un paātrinātu apstiprināšanu. Pēc tam, kad tika publicēti PTC 2. fāzes pētījuma 12 mēnešu starpposma dati, kas liecina, ka
Prilenia, klīniskās stadijas biotehnoloģiju uzņēmums, kas koncentrējas uz steidzamu misiju izstrādāt jaunas terapijas zāles, lai palēninātu neirodeģeneratīvo slimību un neiroloģiskās attīstības traucējumu progresēšanu, ir iesniedzis Eiropas Zāļu aģentūrai (EMA) Eiropas tirdzniecības atļaujas pieteikumu (MAA) attiecībā uz pridopidīnu kā Hantingtona slimības (HD) ārstēšanas līdzekli. MAA
Es piederu jau trešajai Hantingtona slimības skartās ģimenes paaudzei, taču tikai 2024. gadā, pateicoties mana otrā tēva F. Kuka nenogurstošajām pūlēm, mums izdevās noskaidrot, kā šī slimība ienāca ģimenē. Mans vecvectēvs no mātes puses, francūzis vārdā Šarls Novels Katēns (Charles Novel-Catin, 1883-1936),
Abū Dabī, AAE; 2024. gada 19. jūnijs: IROS, Abū Dabī bāzēta līgumpētniecības organizācija (M42 grupas daļa), sadarbojas ar biotehnoloģiju uzņēmumu Prilenia Therapeutics un Starptautisko Hantingtona slimības asociāciju (IHA), lai plānotu pirmo Hantingtona slimības (HD) klīnisko pētījumu AAE un MENA reģionā,
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