HD Focus

Prilenia Therapeutics en partner Ferrer hebben bekendgemaakt dat het Comité voor Geneesmiddelen voor menselijk gebruik (CHMP) van het Europees Geneesmiddelenbureau (EMA) een negatief advies heeft uitgebracht over de toelating van pridopidine voor de behandeling van de ziekte van Huntington (HD).

Hoewel het nieuws teleurstellend is, geven beide bedrijven aan vastbesloten te blijven om pridopidine beschikbaar te maken voor mensen met Huntington. "We blijven geloven in de werking van dit middel en zullen alle mogelijke wegen onderzoeken in overleg met de regelgevende instanties," aldus een woordvoerder van Prilenia.

Pridopidine

A 12-year study reveals how disrupted sleep may predict Huntington’s disease onset, could link to changes in thinking and thought processing, and contribute to nerve damage. Sleep isn’t just rest, it’s a vital brain process for brain health.

C’è un bisogno evidente di dare voce a una categoria invisibile. Ma sempre più indispensabile. C’è un desiderio di denuncia sociale, il supporto di dati-choc non mancano. C’è l’urgenza di colmare un vuoto normativo a tutela di diritti finora non riconosciuti come sostegni economici reali, una formazione obbligatoria anche per medici sulle dinamiche di assistenza, la costituzione di reti psicologiche gratuite in ogni Asl.

L'articolo Caregiver: l’ora di una legge giusta. E si chiedono: “Di noi che ne sarà?” proviene da huntington-onlus.

We learned on July 25, 2025 that Prilenia’s application to the European Medicines Agency’s (EMA) for pridopidine was not accepted for marketing authorization. While this is perhaps not surprising given the data around this drug in clinical trials thus far, it still comes as a great disappointment for the HD community. So what does this […]

Workshop recordings are now streaming on Youtube

The post 40th Annual HDSA Convention Workshop Recordings Now Streaming first appeared on Huntington's Disease Society of America.

July 25, 2025 NAARDEN, Netherlands & WALTHAM, Mass. & BARCELONA, Spain–(BUSINESS WIRE)– Prilenia Therapeutics B.V. and Ferrer today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for […]

An international collaboration between world leaders in Huntington’s disease (HD) that spans both academia and pharmaceutical companies is giving us new insight into how HD progresses. This study has given researchers a detailed timeline of how brain connectivity changes in HD. Using an advanced technique called MIND, researchers traced how brain communication networks shift over […]

The July 2025 edition of EHDN’s newsletter is now online. 

“You’re such a warrior.” People say it with admiration, as if it’s a badge I should wear proudly. But what if I don’t always want to be a warrior? What […]

While the genetic change that causes Huntington’s disease (HD) leads to several problems for cells, researchers believe they could stem from one core issue: the length of the genetic change increasing over time, like a snowball gaining mass as it rolls downhill. This genetic phenomenon, known as somatic instability or somatic expansion, seems to be […]

Luis Aguilera Vera ha 35 anni, è spagnolo, abita a Madrid. Ha perso il padre qualche anno fa, colpito dalla malattia di Huntington, una malattia rara, ereditaria geneticamente, neurodegenerativa e incurabile. Luis ora è un giovane “a rischio".
È di fronte a questa prospettiva che Luis ha deciso di scrivere un libro. Il titolo già ci catapulta al centro della sua vicenda: Cronaca di una sorte annunciata

L'articolo Cronaca di una sorte annunciata proviene da huntington-onlus.

Luis Aguilera Vera ha 35 anni, è spagnolo, abita a Madrid. Ha perso il padre qualche anno fa, colpito dalla malattia di Huntington, una malattia rara, ereditaria geneticamente, neurodegenerativa e incurabile. Luis ora è un giovane “a rischio".
È di fronte a questa prospettiva che Luis ha deciso di scrivere un libro. Il titolo già ci catapulta al centro della sua vicenda: Cronaca di una sorte annunciata

L'articolo Cronaca di una sorte annunciata. Il podcast che legge il libro di Luis Aguilera sulla sua storia e sulla sfida all’Huntington proviene da huntington-onlus.

Smartphones can help scientists calculate a new clinical measure of HD, called the HD Digital Motor Score. Easier data collection could means less clinic trips, fewer people needed for studies, and a better understanding of HD. 

Glia transplantácia u myší – 16.7.2025  Vedci transplantovali zdravé glia bunky do mozgov myší s HCH. Výsledok bol, zlepšenie pohybu, pamäte a predĺženie života.  Originál článku tu. 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 …

Regeneratívne terapie. Čítajte viac »

New research is challenging how we think about treating brain diseases, like Huntington’s disease (HD). A study from the lab of Dr. Steven Goldman shows that transplanting healthy early-stage support cells from humans into the brains of adult mice that model HD improves movement, memory, and even survival. But that’s not all — these cells, […]

After receiving her Huntington’s disease diagnosis, 18-year-old Brea teamed up with her grandmother Leeanne to host their first...

Do you remember playing “Simon Says” as a child? It was fun, silly, and surprisingly tricky. You had to listen carefully and only act when you heard “Simon says”. If you moved at the wrong time, you were out. It turns out that Simon Says isn’t just a playground game. It’s also a helpful way […]

Join us as we walk together in hope, strength and solidarity to support people impacted by Huntington’s disease....

5 июля в Новосибирске прошла Школа здоровья для семей с Болезнью Гентингтона. Это вторая по счёту встреча, которую провели «Редкие Люди» в Новосибирске. Благодаря поддержке Фонда Президентских грантов, в команду… More »

The post Школа семей с болезнью Гентингтона в Новосибирске! appeared first on Центр помощи пациентам с орфанными заболеваниями.

Aktualizácia s 10.7.2025 Ďalšie výsledky preukázali prínos pre redukcii motorických a aj kognitívnych prejavov HCH. Vraj boli hlásené ľuďmi aj zlepšenia v oblasti pamäte a aj zlepšenie problémov s prehĺtaním.  Tento liek bol schválený v USA v roku 2023.  Originál článku tu. Aktualizácia s 29.3.2025 Výsledky III fáze preukázali zníženie mimovoľných pohybov hlavne na rukách a …

Valbenazín – KINECT-HD2 – Ingrezza – Aktualizácia 24.4.2024 Čítajte viac »