SOM Biotech annuncia i risultati dello studio di Fase 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 stipula un accordo globale di licenza e collaborazione con Novartis per il programma PTC518 sulla malattia di Huntington

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 cessa lo sviluppo di dalzanemdor nell'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 annuncia un finanziamento di 35 milioni di sterline per far progredire un nuovo farmaco volto a inibire l'espansione somatica nella malattia di Huntington

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 […]
La Pridopidina di Prilenia per la malattia di Huntington è stata accettata per la revisione dell'autorizzazione all'immissione in commercio in Europa

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 annuncia un aggiornamento positivo dei dati ad interim che dimostrano il rallentamento della progressione della malattia negli studi di Fase I/II di AMT-130 per l'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 annuncia risultati positivi dallo studio di fase 1b/2a SELECT-HD con la prima dimostrazione clinica dell'abbassamento allele-selettivo dell'huntingtina mutante nella malattia di Huntington

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 ha annunciato oggi i promettenti risultati a 12 mesi dello studio di Fase 2 PIVOT-HD su PTC518 orale.

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 […]
Lo studio di fase 2 di Sage Therapeutics rafforza l'impatto cognitivo della malattia di Huntington

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 […]
La FDA concede a uniQure la designazione di terapia avanzata di medicina rigenerativa (RMAT)

Oggi la comunità di Huntington è molto felice. L'annuncio di Uniqure che l'azienda ha ricevuto la "designazione per la terapia genica sperimentale AMT-130" (Regenerative Medicine Advanced Therapy, RMAT) [...]