HD Focus

Empresa de biotecnologia detalha os resultados preliminares de seu medicamento experimental e confirma o Brasil como um dos países na rota do estudo FALCON-HD.

O post Nova fase de testes do SKY-0515 acontecerá no Brasil, anuncia Skyhawk em carta à comunidade apareceu primeiro em ABH – Associação Brasil Huntington.

⏱️7 min read | In 2025, you helped us reach 329,000 people with independent, unbiased HD research news. Our 2025 Annual Report is here. Take a look at what we built together, and consider helping us keep going.

Aktualizácia 4.6.2026 Po 12 mesiacoch fázy I/II. predbežné výsledky hlásia zlepšenie viacerých ukazovateľov ochorenia, zníženie mutovaného HTT proteínu o 69% a zníženie proteínu PMS1 (spoluzodpovedného za somatickú nestabilitu o 26%) o 26%. Nábor do štúdia fázy III. bol ukončený v Austrálii a na Novom Zélande a rozbieha sa celosvetová štúdia Falcon – HD vo fáze …

SKY-0515 – Skyhawk Therapeutics Čítajte viac »

Aktualizácia 13.5.2025 Pri rozbore proteínu a jeho editácii v myšom modeli sa zistilo že kľúčový je malý fragment s názvom HTT1. Jeho znížením oddialili nástup príznakov u myší.   Originál článku tu. Ďalší článok o fragmente HTT1 tu. Aktualizácia 2.8.2025 Vedci robili experimenty s kmeňovými bunkami a dospeli k záveru, že zdravý HTT proteín potrebujeme a preto nie …

HTT proteín a jeho význam Čítajte viac »

SKY-0515_Community Letter_Twelve-Month Phase 1C Patient Data_01JUN26

The post Skyhawk Therapeutics Announces Twelve-Month Interim Results from Phase 1/2 Clinical Trial of SKY-0515 in Huntington’s Disease first appeared on Huntington's Disease Society of America.

<p>The post Skyhawk Therapeutics Announces Twelve-Month Interim Results from Phase 1/2 Clinical Trial of SKY-0515 in Huntington’s Disease first appeared on Huntington's Disease Society of America.</p>

Cabaretier Max van den Burg doet een persoonlijke oproep: vier ons 50-jarig jubileumfeest op zaterdag 13 juni. Het belooft een bijzondere dag te worden vol ontmoeting, muziek en natuurlijk een feestelijk tintje. Voor leden, families en Huntington professionals.

 

Aanmelden kan hier!

 

Zeldzaam, maar je staat niet alleen 🩷💚

 

---

 

SKY-0515_Community Letter_Twelve-Month Phase 1C Patient Data_01JUN26

The post Community letter: Skyhawk Therapeutics Announces Twelve-Month Interim Results from Phase 1/2 Clinical Trial of SKY-0515 in Huntington’s Disease first appeared on Huntington's Disease Society of America.

<p>The post Community letter: Skyhawk Therapeutics Announces Twelve-Month Interim Results from Phase 1/2 Clinical Trial of SKY-0515 in Huntington’s Disease first appeared on Huntington's Disease Society of America.</p>

Registration is open for the EHDN Clinical Research Congress, which this year will be held in Krakow, Poland, from 22 […]

Krakow, Poland | October 22–24, 2026

Um ano após o início do tratamento, o medicamento experimental segue demonstrando melhora clínica, redução da huntingtina mutante e de proteína associada à expansão da doença. Estudo maior está em fase de recrutamento.

O post Skyhawk apresenta resultados de Fase 1/2 do SKY-0515 após doze meses de estudo apareceu primeiro em ABH – Associação Brasil Huntington.

One year into treatment, SKY-0515 continues to lower huntingtin and shows encouraging trends with signs and symptoms of HD. A larger trial is enrolling to determine whether the drug truly slows HD progression

<p>The post Skyhawk Therapeutics Announces Twelve-Month Interim Results from Phase 1/2 Clinical Trial of SKY-0515 in Huntington’s Disease first appeared on Huntington's Disease Society of America.</p>

⏱️8 min read | May 2026: 2 year data from the votoplam trial suggests disease progression may slow; AMT-130 heads to the UK for regulatory review; pigs reveal immune cells invading the HD brain; and new science zooms in on toxic huntingtin fragments.

For Huntington’s Disease Awareness Month, Eddie’s story is one of fierce love, resilience, and determination – not just to care […]

Comissão mantém parecer desfavorável à inclusão do medicamento no sistema público de saúde fundamentando a decisão no alto impacto orçamentário e na incerteza do benefício clínico em longo prazo.

O post Conitec rejeita incorporação da deutetrabenazina para doença de Huntington no SUS apareceu primeiro em ABH – Associação Brasil Huntington.

Francophone Youth Day – Written by the Moving Forward team on 27th May 2026 On April 25, the Association Huntington France (AHF) hosted its first Francophone…

Raising Huntington’s Disease Awareness: Beyond the Month of May As May draws to a close and Huntington’s Disease (HD) Awareness Month ends, advocates are urging the public to continue the fight for awareness and support year-round. For those who do not know, Huntington’s Disease—a rare, inherited neurodegenerative disorder—affects thousands of families across the United States, yet remains widely misunderstood. Organizations like Help 4 HD International are leading the charge to keep HD in...

⏱️7 min read | In a recent paper, scientists targeted a small, harmful piece of the huntingtin message (HTT1a) in HD mice. This reduced toxic protein clumps and delayed genetic changes, more than targeting full length HTT.