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 Baca lebih lanjut...
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 Baca lebih lanjut...
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 Baca lebih lanjut...
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 Baca lebih lanjut...
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 Baca lebih lanjut...
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 Baca lebih lanjut...
PTC518 adalah obat oral yang mengurangi produksi protein Huntingtin yang bermutasi yang menyebabkan perkembangan penyakit. Program dengan penunjukan Jalur Cepat dapat memperoleh manfaat dari interaksi awal dengan FDA, dan mungkin memenuhi syarat untuk tinjauan prioritas dan persetujuan yang dipercepat. Hal ini menyusul dirilisnya data sementara selama 12 bulan dari studi Fase 2 PTC, yang Baca lebih lanjut...
Prilenia, sebuah perusahaan bioteknologi tahap klinis yang berfokus pada misi mendesak untuk mengembangkan terapi baru untuk memperlambat perkembangan penyakit neurodegeneratif dan gangguan perkembangan saraf, telah mengajukan Permohonan Otorisasi Pemasaran Eropa (MAA) kepada European Medicines Agency (EMA) untuk pridopidin sebagai pengobatan penyakit Huntington (HD). Sebuah MAA Baca lebih lanjut...
Abu Dhabi, UEA; 19 Juni 2024: IROS, sebuah organisasi penelitian kontrak yang berbasis di Abu Dhabi (bagian dari grup M42), telah bermitra dengan Prilenia Therapeutics, sebuah perusahaan bioteknologi tahap klinis, dan International Huntington's Disease Association (IHA) untuk merencanakan uji klinis pertama untuk penyakit Huntington (HD) di UEA dan wilayah MENA, Baca lebih lanjut...
"Kami sangat senang dengan data baru ini yang menunjukkan perlambatan perkembangan penyakit Huntington yang signifikan secara statistik dan bergantung pada dosis serta penurunan NfL di CSF pada 24 bulan," kata Walid Abi-Saab, M.D., kepala petugas medis uniQure. "Kami percaya ini adalah uji klinis pertama dari setiap obat investigasi Baca lebih lanjut...
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