HD Focus

Aktualizácia z 19.4.2025  Testovanie lieku pokračuje v novej štúdii a tento týždeň bola upravená dávka ktorá sa počas štúdie používa.Celkovo je v štúdii 301 účastníkov a dostávajú dávku každých 16 týždňov. Podľa nezávislej komisie asi prináša liek nejaký benefit nakoľko upravili dávkovanie počas štúdie. Testovanie by malo byť ukončené v roku 2026. Originál článku tu. …

Tominersen – Znovuotvorenie programu Čítajte viac »

This week, we heard an update from Roche about their huntingtin-lowering therapy, tominersen, currently being tested in the GENERATION HD2 trial. An independent data monitoring committee (iDMC) that regularly reviews all of the data from the trial recently held their scheduled meeting and made a recommendation to modify the trial design. To cut to the chase and set everyone’s mind at ease - the trial is continuing and there are no major safety concerns. Tominersen still appears to be well tolerated but there are some changes to the trial in regards to the dosing of the drug that the community should know about. Let’s get into it.

Recap - what is tominersen and how does it work?

Tominersen is an experimental drug developed by Roche that is designed to treat Huntington’s disease (HD) by targeting the root cause of the condition: the huntingtin (HTT) gene. People with HD inherit a version of this gene with a mutation at the beginning that leads to the production of a faulty version of the HTT protein. Making this faulty protein is thought to cause damage to brain cells, leading to the progressive symptoms of HD.

HUNTINGTON’S DISEASE SOCIETY OF AMERICA AWARDS MORE THAN $1.8 MILLION TO EXPAND HDSA CENTERS OF EXCELLENCE NETWORK TO SIXTY-NINE SITES ...

The post HUNTINGTON’S DISEASE SOCIETY OF AMERICA AWARDS MORE THAN $1.8 MILLION TO EXPAND HDSA CENTERS OF EXCELLENCE NETWORK TO SIXTY-NINE SITES first appeared on Huntington's Disease Society of America.

~ 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 […]

LEXINGTON, Mass. and AMSTERDAM, April 17, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing...

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 […]

Vi har glädjen att bjuda in er till årets Nationella möte som går av stapeln 22 oktober på hotell Kusten i Majorna, Göteborg. I år har man möjlighet att både delta på plats eller via länk. OBS! Anmälan krävs till båda. Läs mer om tider och anmälan nedan. Antal platser på plats i Göteborg är…

Inlägget Nationellt möte 22 oktober! Läs mer och anmäl dig här! publicerades först på RHS Riksförbundet Huntingtons Sjukdom.

Een mooie mijlpaal! Tien jaar geleden werd het eerste Huntingtoncafé gehouden; een periodiek samenzijn in een informele sfeer waarbij een spreker een bepaald thema uitdiept en er vragen over beantwoordt.

Inmiddels zijn er door het hele land Huntingtoncafés, aangeboden door de verschillende Regionale Expertisecentra. De bijeenkomsten zijn een plek geworden voor ontmoeting, herkenning en ondersteuning. In het Kontaktbkad van september vertelt Jesseke de Man, de bedenkster van het Huntingtoncafé, meer over het idee erachter en wat het patiënten oplevert.

Välkommen att delta på ett stödgruppsmöte i Stockholm lördagen 17 maj. Läs mer om programmet och hur du anmäler dig här nedan! Hoppas vi ses!  

Inlägget Välkommen på stödgruppsmöte i Stockholm 17 maj publicerades först på RHS Riksförbundet Huntingtons Sjukdom.

Sabato 12 aprile si è tenuta l’annuale assemblea degli Associati di Huntington Onlus, un’occasione per rivedersi in collegamento da diverse città italiane, condividere le piste di lavoro che si stanno affrontando, passando per un bilancio dell’anno passato. Grazie Lorenzo! L’apertura dell’incontro è stata affidata al ricordo – letto dalla nostra Presidente Elisabetta CALETTI – del […]

L'articolo I soci di Huntington Onlus in Assemblea – Incontro 12 aprile  proviene da huntington-onlus.

Huntington’s disease (HD) is caused by extra repeats of the DNA letters CAG within the genetic code of the huntingtin gene. We used to think that those CAG lengths were stable in most tissues, but we now have a growing understanding that CAG instability contributes to HD. Somatic instability is the concept that CAG repeats […]

When it comes to discussing difficult topics in relationships, the conversation surrounding Huntington's disease (HD) can be particularly...

We often think of the adult brain like a completed jigsaw puzzle—once all the pieces are in place, that’s it. If a few pieces go missing, as happens in neurodegenerative diseases like Huntington’s disease (HD), there’s not much we can do except try to slow the loss. But new research is challenging that idea in a big way. A new study has shown that it may be possible to grow new brain cells in the adult brain—and not just any cells, but the exact pieces that HD takes away. Even more amazing? These new cells can connect with the brain’s existing networks, as if finding their place in the puzzle and clicking right into place. This discovery opens the door to a bold new goal: not just slowing the loss, but rebuilding the puzzle itself.

What Gets Lost in Huntington’s Disease?

To see why this study matters, let’s start with the pieces that go missing in HD. The disease causes progressive damage to an area of the brain called the striatum. The striatum sits almost exactly in the center of the head and helps control movement, emotions, and decision-making. The specific puzzle pieces lost here are called medium spiny neurons, or MSNs.

Podarilo sa obnoviť a začleniť nové neuróny do poškodených mozog myší – 8.4.2025Toto sa podarilo použitím vlastných buniek myší a ich stimuláciou. Pomocou dvoch molekúl sa im podarilo spustiť v mozgu generovanie nových neurónov, ktoré sa napojili na stávajúcu mozgovú štruktúru. Molekuly sa podali priamo do mozgu myší. Táto terapia zlepšila pohyblivosť a stlmila mimovoľné …

Kmeňové Bunky – transplantácia Glia buniek Čítajte viac »

Sabato 17 maggio, nella Sala Mediateca dell’I.S.S.R. in Via Nomentana 56, sede di A.I.C.H.-Roma OdV, avrà luogo il nostro ventinovesimo convegno, che è anche il primo del 2025. In questo incontro di maggio, abbiamo pensato di approfondire gli argomenti presentati dalla Prof.ssa Anna Rita Bentivoglio – i 14 punti per avere un cervello più sano- […]

L'articolo EFFICACIA TERAPEUTICA DELLA RETE DI RELAZIONI NELLA MH sembra essere il primo su corea di huntington -AICH ROMA ONLUS -.

Join us on Monday, 12 May for a special Community and Online Forum hosted by Skyhawk Therapeutics, where...

Podarilo sa obnoviť a začleniť nové neuróny do poškodených mozog myší – 8.4.2025 Toto sa podarilo použitím vlastných buniek myší a ich stimuláciou. Pomocou dvoch molekúl sa im podarilo spustiť v mozgu generovanie nových neurónov, ktoré sa napojili na stávajúcu mozgovú štruktúru. Molekuly sa podali priamo do mozgu myší. Táto terapia zlepšila pohyblivosť a stlmila …

Regeneratívne terapie. Čítajte viac »

Il 17 e 18 marzo si è tenuto al Palacongressi di Rimini il Convegno delle Associazioni in rete di Telethon, dal titolo: “La strada della ricerca dal laboratorio alla persona con malattia genetica rara: un percorso dove ogni passo conta”. L’evento, giunto all’VIII edizione, ha visto la partecipazione di circa 500 ricercatori e oltre 200 […]

L'articolo Huntington Onlus al Convegno Telethon 2025 proviene da huntington-onlus.

Imagine battling a disease that not only affects your body but also causes your mind to lose touch with reality, making it hard to see the world as it really is. This is the heartbreaking reality for many people living with Huntington’s disease (HD) who also experience symptoms of psychosis. Professor Clement Loy and his […]