HD Focus

In an update today, Skyhawk Therapeutics announced that the first participants in its United States study trial for...

Skyhawk’s FALCON-HD trial has dosed the first participants in the US with the drug, SKY-0515. The once-daily pill targets two drivers of HD, but does it actually slow disease? That’s the big question this study hopes to answer.

Skyhawk anuncia as primeiras doses do SKY-0515 nos EUA. O comunicado não traz novos dados de eficácia

O post FALCON-HD: primeiros participantes recebem doses do medicamento experimental nos EUA apareceu primeiro em ABH – Associação Brasil Huntington.

V celej Európe sa rozhoduje o liečbe Huntingtonovej choroby a ľudia, ktorí s touto chorobou žijú každý deň, do týchto rozhodnutí mohli hovoriť len veľmi málo. European Huntington Association spolu s European Federation of Neurological Associations realizuje dotazník, ktorý to má zmeniť. Veľmi nám záleží na vašej odpovedi. Pýtame sa, ktoré príznaky vás zasahujú najviac, …

Čo je pre vás pri Huntingtonovej chorobe najdôležitejšie? Čítajte viac »

From sticky thoughts and pigs that model HD to DNA repair and promising drug trial results, October brought fresh insights into HD. Here’s what you might have missed!

uniQure Announces FDA Acceptance and Priority Review of the Biologics License Application for ifezuntirgene inilparvovec for the

The post UniQure Announces FDA Acceptance and Priority Review of the Biologics License Application for ifezuntirgene inilparvovec for the first appeared on Huntington's Disease Society of America.

U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for ifezuntirgene inilparvovec (eye-feh-ZUN teer-JEEN in-il-par-VOE-vek) (also referred to as AMT-130), an investigational gene therapy candidate for the treatment of Huntington’s disease, for review

The FDA has accepted uniQure’s application for AMT-130, an experimental gene therapy for Huntington’s disease, and set a May 2027 decision date. Here’s what this milestone means, and what still needs to happen.

2026 Nov 2 uniQure Regulatory BLA Update Community Letter_Final

The post UniQure Regulatory BLA Update Community Letter first appeared on Huntington's Disease Society of America.

Quatro centros no Brasil estão recrutando pessoas com Doença de Huntington para o estudo FALCON-HD. Veja a situação de cada centro e como participar

O post Atualização dos centros de pesquisa do estudo FALCON-HD apareceu primeiro em ABH – Associação Brasil Huntington.

An experimental treatment for HD developed by Alnylam has shown long-lasting huntingtin lowering after just one dose, with encouraging early safety data. A pivotal trial is now planned for 2027 to figure out if this drug, brezosiran, can slow HD.

Journalist Carlos Briceno’s vrouw Jill heeft de ziekte van Huntington. Carlos deelt zijn ervaringen in een column. Onlangs schreef hij over de vraag die hij van zijn vrouw kreeg: “Wat als het medicijn erger is dan de ziekte?”

Mijn vrouw Jill praat meer over de toekomst dan ze vroeger deed. Het zijn geen gesprekken over vakanties of pensioenplannen, maar een stiller en zwaarder gesprek dat hoort bij het gendrager zijn van de ziekte van Huntington. Onlangs keek ze me aan vanaf het uiteinde van de keukentafel en vroeg: "Op welk punt wordt proberen iemand in leven te houden erger dan de ziekte zijn gang laten gaan?"

Het was geen theoretische vraag. Het ging over haar en over ons.

Huntington is een progressieve ziekte en Jill weet hoe dat verloop eruit kan zien. Haar vader, samen met meerdere familieleden aan zijn kant van de familie, waren eveneens gendrager.

Desde el pasado 23 de octubre de 2026 está en vigor la Ley 4/2026, de 1 de octubre, que introduce importantes modificaciones en la legislación española sobre discapacidad y dependencia. […]

La entrada La nueva Ley de Discapacidad y Dependencia ya está en vigor: principales novedades se publicó primero en .

29.10.2026 Podľa najnovších výsledkov liek dostal označenie Brezosiran  a znižuje hladiny mutovaného HTT proteínu. Začiatok hlavnej časti štúdie sa plánuje na budúci rok. Terapia sa podáva lumbálnou punkciou a štúdia sa plánuje v UK, USA a Nemecku. Zatiaľ bola podaná jedna dávka a po 3 mesiacoch mali pacienti, ktorý dostali vysokú dávku lieku znížený HTT …

Alnylam – ALN-HTT02 Čítajte viac »

October offered plenty of promising news and innovative approaches in Huntington’s disease research around the world. This month,...

L’autunno porta con sé un appuntamento a cui teniamo moltissimo: il nostro trentaduesimo convegno, dal titolo “Un viaggio nella malattia di Huntington: dalle teorie più avanzate della ricerca ai consigli pratici nell’assistenza quotidiana al malato”. Le nostre relatrici ci accompagneranno in un percorso a due tappe. Da un lato, le ultime notizie dal mondo della […]

L'articolo AICH Roma: il 32° convegno sulla malattia di Huntington sembra essere il primo su corea di huntington -AICH ROMA ONLUS -.

Dal 22 al 24 ottobre Cracovia ha ospitato l’EHDN Clinical Research Congress 2026, il grande appuntamento europeo dedicato alla malattia di Huntington. Ricercatori, clinici, associazioni e familiari si sono ritrovati per confrontarsi sullo stato della ricerca, sulle terapie in sviluppo e sulle prospettive per il futuro. Accanto agli aggiornamenti scientifici, ampio spazio è stato dedicato […]

L'articolo Dal congresso EHDN di Cracovia: ricerca e assistenza al centro sembra essere il primo su corea di huntington -AICH ROMA ONLUS -.