HD Focus

Autumn is here, and with it comes a fresh edition of HA Highlights — our community newsletter that...

Science and patient support go hand in hand: Poland has joined the Moving Forward project – Written by the Moving Forward team on 6th March 2026…

Update from the Huntington’s Disease Society of America (HDSA)  The Huntington’s disease (HD) community currently has no approved disease-modifying therapies. While...

The post Update from the Huntington’s Disease Society of America (HDSA)  first appeared on Huntington's Disease Society of America.

<p>The post Update from the Huntington’s Disease Society of America (HDSA)  first appeared on Huntington's Disease Society of America.</p>

De Amerikaanse Food and Drug Administration (FDA) heeft biotechnologiebedrijf uniQure gevraagd om een extra klinische studie uit te voeren. Dit moet gebeuren voordat het bedrijf een aanvraag kan indienen voor de goedkeuring van AMT-130, een experimentele gentherapie voor de ziekte van Huntington.

Dit nieuws volgt op een officieel overleg tussen uniQure en de FDA op 30 januari, waarbij het traject naar goedkeuring werd besproken. Eind vorig jaar liet de toezichthouder al weten dat de huidige positieve resultaten uit lopende Fase 1/2-onderzoeken in de VS en Europa niet voldoende zijn als basis voor een directe markttoelating.

Katie Jackson's response can be read at the following link: https://news.bloomberglaw.com/pharma-and-life-sciences/fda-push-for-10-hour-fake-brain-surgery-trial-raises-concerns

ATTENZIONE POSSIBILITA’ DI ERRORI Per garantire una diffusione rapida e capillare delle notizie relative alla ricerca sull’HD e agli aggiornamenti sulle sperimentazioni in corso, questo articolo è stato sintetizzato tramite Intelligenza Artificiale dal sito HDBUZZ.NET e non è stato  revisionato da un redattore umano. Per una consultazione più affidabile, si consiglia di fare riferimento alla […]

L'articolo Occhi come finestra sul cervello: i biomarcatori oculari nella malattia di Huntington sembra essere il primo su corea di huntington -AICH ROMA ONLUS -.

⏱️ 8 min read | An oral Huntington's disease drug is eligible for accelerated approval in Australia. This isn't full approval yet, but it opens a faster path to potentially getting this once-daily pill to people with HD sooner.

For ANY questions: medinfo@uniqure or 1-866-520-1257

Prehľad o nových postupoch so Slovenskými titulkami. https://player.vimeo.com/video/1084349951?texttrack=sk&badge=0&autopause=0&player_id=0&app_id=58479&fbclid=IwY2xjawQSe6xleHRuA2FlbQIxMQBzcnRjBmFwcF9pZBAyMjIwMzkxNzg4MjAwODkyAAEe3G37nIPXveNhcmmbgi_J9o4BfnGZx-O80e7tAAb6ne2VbOCYmarZa7ivoM4_aem_hDAL-1BGdMXmmPSPSD5G_g

Dear Huntington’s Disease Community, We are writing to share an update following our recent Type A meeting with the U.S. Food and Drug Administration (FDA.) On March 2, 2026, uniQure […]

⏱️ 10 min read | The FDA wants more data before approving AMT-130 for Huntington’s disease in the U.S. On March 2026, uniQure shared in an update that current Phase 1/2 data weren’t enough for the agency. A new randomized, sham-controlled trial may be required.

<p>The post uniQure Regulatory Update Community Letter first appeared on Huntington's Disease Society of America.</p>

aktualizácia 2.3..2026 Pridopidín v kombinácii s HA10 znížil stratu neurónov buniek až o 74% Originál článku tu. aktualizácia z 17.10.2025 Podľa dvojročných výsledkov Pridopidín spomaľuje progres ochorenia u ľudí ktorý neužívajú antidopamínové lieky. Práve na tejto skupine ľudí plánuje Prelinea a Ferrera budúci rok rozbehnúť doplnkovú štúdiu vo fáze III. Podľa rozboru bolo spomalenie progresu ochorenia …

Pridopidin Čítajte viac »

Vi har uppmärksammat ett problem med inbetalningar av medlemsavgiften. OCR-numret fungerar för närvarande inte i OCR-fältet, utan behöver i stället anges i meddelandefältet! För frågor kontakta info@huntington.se

Inlägget Problem med medlemsavgift! dök först upp på RHS Riksförbundet Huntingtons Sjukdom.

⏱️ 6 min read | Vico Therapeutics’ Huntington’s disease drug VO659 is now being tested just twice a year, and the FDA has cleared the way for US trials to begin later this year.

I dag, den 28 februari, uppmärksammar vi Sällsynta dagen. Bakom varje sällsynt diagnos finns en människa, en familj och en berättelse.Idag lyfter vi röster från familjer som lever med Huntingtons sjukdom – röster som alltför sällan får höras i samhället. Häromveckan publicerade vi en artikel om Huntingtons sjukdom i DN- för att öka allmänhetens kunskap…

Inlägget Sällsynta dagen 28 februari dök först upp på RHS Riksförbundet Huntingtons Sjukdom.

FDA Seeks Feedback from Rare Disease Community The U.S. Food and Drug Administration (FDA) has opened a public docket to solicit input from the rare disease community. The FDA docket, available here , invites public comments from parties engaged in the design and conduct of rare disease clinical trials, including sponsors, investigators, patients, and patient advocates. This docket includes two distinct opportunities for feedback: The Center for Drug Evaluation and Research’s Accelerating...

Evento promovido pelo Muitos Somos Raros e Casa Hunter marcou o lançamento da campanha "Doença Rara Não Tem Idade", iniciativa que conta com o apoio da nossa Associação.

O post ABH participa de coletiva de imprensa e lançamento de campanha de doenças raras em São Paulo apareceu primeiro em ABH – Associação Brasil Huntington.

⏱️ 8 min read | February 2026 HD research recap: published trial data from branaplam trial; new insights into the genetics of the HD "grey zone"; plus worms, brain circuits, eye tracking, and the hidden psychological weight of living at risk.

Voluntárias marcaram presença em atividade que destacou os desafios no acesso à saude enfrentados por famílias e pacientes.

O post ABH participa de encontro sobre doenças raras na Câmara Municipal de Porto Alegre apareceu primeiro em ABH – Associação Brasil Huntington.