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 Authorization Application (MAA) with the European Medicines Agency (EMA) for Leggi tutto...
I belong to the third generation of a family affected by Huntington’s Disease, but it wasn’t until 2024, thanks to the tireless efforts of my second father, F. Cook, that we were able to confirm how the disease entered the Leggi tutto...
Abu Dhabi, UAE; June 19, 2024: IROS, an Abu Dhabi-based contract research organization (part of the M42 group), has partnered with Prilenia Therapeutics, a clinical-stage biotech company, and the International Huntington’s Disease Association (IHA) to plan the first-ever clinical trial Leggi tutto...
“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,” stated Walid Abi-Saab, M.D., chief medical officer of uniQure. “We believe Leggi tutto...
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) protein and preserves healthy, wild-type huntingtin (wtHTT) protein for individuals Leggi tutto...
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 mHTT was lowered by 21% and 43% respectively, for 5mg Leggi tutto...
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 between 29 healthy volunteers and 40 participants with HD. As Leggi tutto...
Il mese scorso, il presidente dell'IHA Svein Olaf Olsen ha tenuto un discorso sulla malattia di Huntington al MENA Congress for Rare Diseases di Abu Dhabi. Ha parlato dell'urgente necessità di istituire un centro di eccellenza per le malattie neurodegenerative nella regione e Leggi tutto...
Oggi la comunità Huntington è molto felice. L'annuncio di Uniqure di aver ricevuto la "designazione" di Regenerative Medicine Advanced Therapy (RMAT) per la terapia genica sperimentale AMT-130 nella malattia di Huntington è più che promettente", afferma il Presidente di Uniqure. Leggi tutto...
Nel suo libro di memorie "We Exist", Tanita Allen racconta le difficoltà incontrate nel far diagnosticare correttamente i suoi sintomi come malattia di Huntington. Più volte le è stata fatta una diagnosi errata perché i medici si sono rifiutati di considerare che anche gli afroamericani potevano ereditare la malattia mutata. Leggi tutto...
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