Kategorija: Raziskave

UniQure sporoča, da je FDA podelila imenovanje za prebojno terapijo AMT-130 za zdravljenje Huntingtonove bolezni

~ Breakthrough Therapy designation based on clinical evidence from Phase I/II trials showing meaningful slowing of disease progression ~ ~ Additional regulatory update and guidance on the Biologics License Application […]

Posodobitev študije faze II GENERATION HD2

Roche has announced the continuation of the GENERATION HD2 Phase II clinical trial evaluating tominersen in individuals with early signs of Huntington’s disease. Following an interim analysis by an independent committee, no […]

Družba SOM Biotech objavi rezultate študije faze 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, […]

Družba PTC Therapeutics sklene globalni licenčni sporazum in sporazum o sodelovanju z družbo Novartis za program PTC518 za Huntingtonovo bolezen

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 […]

Družba Sage Therapeutics ustavi razvoj dalzanemdorja pri 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 […]

Družba LoQus23 Therapeutics je napovedala financiranje v višini 35 milijonov funtov za razvoj novega zdravila za zaviranje somatske širitve pri Huntingtonovi bolezni

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 […]

Pridopidin družbe Prilenia za Huntingtonovo bolezen sprejet za pregled evropskega dovoljenja za promet

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 objavlja pozitivne vmesne podatke, ki dokazujejo upočasnitev napredovanja bolezni v I/II fazi preskušanja AMT-130 za 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,” […]

Družba Wave Life Sciences napoveduje pozitivne rezultate preskušanja faze 1b/2a SELECT-HD s prvim kliničnim dokazom alelno selektivnega znižanja vrednosti mutiranega huntingtina pri Huntingtonovi bolezni

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) […]

Družba PTC je danes objavila obetavne 12-mesečne rezultate študije faze 2 PIVOT-HD o peroralnem zdravilu 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 […]