Categoria: Notizie

UniQure annuncia che la FDA ha concesso la designazione di terapia innovativa ad AMT-130 per il trattamento della malattia di Huntington

~ 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 […]

Aggiornamento sullo studio di fase 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 […]

Uno sguardo indietro al Congresso HDYO 2025

The HDYO Congress 2025 brought together young people, families, researchers, and professionals from around the world for an unforgettable event dedicated to the Huntington’s Disease community. Over three inspiring days, […]

23 marzo: celebrazione della Giornata della Gratitudine nella comunità della Malattia di Huntington

“Gratitude Day is a way to honor the collaboration of HD families with clinicians and scientists, which started with the efforts to identify the Huntington’s disease gene in the 1980s, […]

Be Empowered di Tanita Allen, la nuova rubrica del sito IHA

Tanita Allen, a dedicated advocate for Huntington’s Disease and author of her much labored memoir “We Exist”, is starting a new column for the IHA website, called Be Empowered by […]

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, […]

Campagna per la Giornata delle malattie rare 2025

The International Huntington Association, in collaboration with the European Huntington Association, is organizing a campaign for Rare Disease Day 2025, on the 28th February. The slogan for this year’s RDD […]

RDI lancia il programma di leadership giovanile

Rare Diseases International (RDI), announced the launch of the RDI Youth Leadership Programme, an initiative designed to engage and empower young people within the rare disease community. This programme aims […]

Dodicenne pubblica un libro per bambini sulla malattia di Huntington

Toronto student writes book so others will treat her grandpa kindly December 11, 2024 — Toronto, Ontario, Canada Most people have never heard of Huntington’s disease. This lack of awareness […]

UniQure annuncia l'allineamento con la FDA sugli elementi chiave del percorso di approvazione accelerata per AMT-130

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 […]