SOM Biotech ogłasza wyniki badania fazy 2b

SOM Biotech presents the Phase 2b study results with SOM3355 demonstrating a unique profile with robust improvements of chorea in Huntington’s Disease Patients and a safe profile with no somnolence, […]
PTC Therapeutics zawiera globalną umowę licencyjną i umowę o współpracy z Novartis w zakresie programu PTC518 dotyczącego choroby Huntingtona

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 […]
Sage Therapeutics zaprzestaje rozwoju dalzanemdoru w HD

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 […]
LoQus23 Therapeutics ogłasza finansowanie w wysokości 35 milionów funtów na rozwój nowego leku mającego na celu zahamowanie ekspansji somatycznej w chorobie Huntingtona.

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 […]
Pridopidyna firmy Prilenia stosowana w chorobie Huntingtona dopuszczona do europejskiego przeglądu pozwoleń na dopuszczenie do obrotu

Prilenia, a clinical stage biotechnology company focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders, has filed a European Marketing […]
uniQure ogłasza pozytywną aktualizację danych okresowych wykazujących spowolnienie progresji choroby w badaniach fazy I/II AMT-130 dla HD

“We are very pleased with these new data demonstrating a statistically significant, dose-dependent slowing of the progression of Huntington’s disease and lowering of NfL in the CSF at 24 months,” […]
Wave Life Sciences ogłasza pozytywne wyniki badania fazy 1b/2a SELECT-HD, w którym po raz pierwszy wykazano kliniczne działanie obniżające stężenie zmutowanej huntingtyny w chorobie Huntingtona.

Today, Wave Life Sciences announced positive results from SELECT-HD, our Phase 1b/2a placebo-controlled trial evaluating the investigational therapy WVE-003. These results demonstrate that WVE-003 selectively lowers toxic, mutant huntingtin (mHTT) […]
PTC ogłosiła dziś obiecujące 12-miesięczne wyniki badania fazy 2 PIVOT-HD doustnego PTC518

At Month 12, mutant Huntingtin (mHTT) in the blood was lowered by 22% and 43% respectively for 5mg and 10mg doses. A similar result was seen in cerebrospinal fluid, where […]
Badanie fazy 2 firmy Sage Therapeutics potwierdza wpływ choroby Huntingtona na funkcje poznawcze

Earlier this week Sage Therapeutics announced the results of their SURVEYOR study which quantified the cognitive impact of Huntington’s Disease. Using the HD-Cognitive Assessment Battery (HD-CAB) they measured the difference […]
FDA przyznaje uniQure Regenerative Medicine Advance Therapy (RMAT) Designation.

Today we are very happy in the Huntington community. The announcement from Uniqure that the company has received the Regenerative Medicine Advanced Therapy (RMAT) “Designation for Investigational Gene Therapy AMT-130 […]