HD Focus: News Across the Globe

Disclaimer: I have written this piece from a position of privilege - as an HD family member that has been fortunate to receive an education that allows me to deeply understand the nuances of Huntington’s disease. I know what it means not only at the biological level, but also at the family level. I am profoundly aware of the desire for a disease-modifying drug. But my hopes are tempered through the privileged lens of understanding complex scientific data and interpretation. Here, I report facts and my opinion of those facts with no vested interest in any specific therapeutic approach. If Prilenia feels errors have been made, they are invited to reach out and any factual corrections will gladly be made.

Recently, there have been a few press releases from Prilenia Therapeutics about their advancement of the drug pridopidine toward regulatory approval for the treatment of Huntington’s disease (HD). There’s also been mixed messaging about findings from pridopidine clinical trials and statements made by the company. Let’s break down what the research really says and what the recent press releases mean in the bigger picture.

MermaiHD Trial

Prilenia včera požiadala o schválenie Pridopidínu aby bolo možné ho predávať v rámci Európy. EMA túto žiadosť vyhodnotí v priebehu 12 – 14 mesiacov.  Potom bude nasledovať schvaľovanie v jednotlivých štátoch aby bol liek dostupný. Originál článku tu. aktualizácia z 4.9.2024 Prilenia požiada v polovici roku 2024 o schválenie Pridopidínu, Európskou liekovou agentúrou (EMA). Ak …

Pridopidin Čítajte viac »

Houston accountant Margaret Moncrieff (79) is taking on her 13th Walk of Hope in support of families, including her own, who […]

Добрый день!14 сентября в 12.00 часов состоится Информационный (поликлинический) день для семей с болезнью Гентингтона .На инфо дне вы получите бесплатные консультации у лучших специалистов в области изучения и лечения… More »

The post Информационный ( поликлинический) день 14 сентября 2024 г. appeared first on Центр помощи пациентам с орфанными заболеваниями.

Prilenia, a clinical stage biotechnology company focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders, has filed a European Marketing […]

Utbildningsdagen riktar sig framför allt till omvårdnadspersonal och anhöriga, men alla är välkomna. Vi diskuterar omvårdnad – lär känna igen svåra komplexa symtom, etiska dilemman, kommunikation, medicinering och anhörigperspektiv. Vi eftersträvar en samsyn för att säkerställa omvårdnadsbehovet för vår målgrupp oavsett var man bor i landet och var man får sin vård. Skriv ut programmet…

Inlägget Anmäl dig nu till utbildningsdag ” Palliativ omvårdnad” – 27 september (dagen efter Nationellt mötet). publicerades först på RHS Riksförbundet Huntingtons Sjukdom.

RHS har nöjet att bjuda in dig som är ung vuxen till en helg i Karlstad den 18-20 oktober! Läs mer och anmäl dig nedan! Hoppas vi ses!    

Inlägget Inbjudan till helg för unga vuxna i Karlstad 18-20 oktober publicerades först på RHS Riksförbundet Huntingtons Sjukdom.

Deň 1 : Prvá bola prednáška o CRISPR technológii a jej možnom použití pri HCH. Druhá prednáška doktora Eda Wilda bola o štúdiách v úvode spomenul Tominersen od Roche. Nasledoval PTC-518 od PTC therepeutics – je to malá molekula, podávaná formou tablety a znižujúca HTT podľa dávky lieku.  Liek sa posúva do 3 fázy testovania. Ďalším …

HDF konferencia 2024 Čítajte viac »

Di seguito il nostro periodico con interessanti aggiornamenti della ricerca uniQure, di cui vi aveva parlato la nostra cara Marina Frontali nel convegno del 18 maggio 2019. Oltre, alla interessante traduzione della dott.ssa Petrucci sulla ricerca uniQure, vi troverete informazioni sul prossimo congresso EHDN che si terrà a Strasburgo prossimamente. Il convegno AICH-Roma si terrà […]

L'articolo PERIODICO MAGGIO – AGOSTO sembra essere il primo su corea di huntington -AICH ROMA ONLUS -.

In Pakistan, Huntington’s disease is often misunderstood and a burden that many families bear alone without any support. Our portraits of resilience series aims to showcase the grit and resilience of the Pakistani HD community facing an incurable disorder in a country where services for rare disease are nearly nonexistent. Our first post is an […]

Como vimos en nuestro artículo anterior, la Enfermedad de Huntington (EH) es una condición genética hereditaria que plantea serios desafíos para las personas que desean formar una familia.  Dado que […]

La entrada Planificación Familiar en la Enfermedad de Huntington: Opciones Reproductivas y Consideraciones Importantes (Parte 1) se publicó primero en .

Stem cells grown in 3D in a research lab can mimic some features of Huntington’s disease (HD). They also hold promise for transplantation studies to potentially add back cells that are lost in HD. But what would happen to those new cells? Would they get along with the cells still in the brain that have the HD gene? And what can this system teach us about ongoing clinical trials aimed at lowering the HD-causing message in only parts of the brain? Read on to find out!

The power of stem cells

Stem cells hold a certain mystique. They can either retain their “stemness”, remaining a stem cell, or to turn into something else altogether. Contained within each one is the ability to become almost any cell type in the human body. Scientists can coax them into becoming a heart cell, or a muscle cell, or even a brain cell, providing scientists with a powerful research tool that can be used to answer questions about people’s brains in health and disease.

This year’s Family Gathering will take place on Saturday 9 November at Apex City Quay Hotel, Dundee. This year, in […]

No dia 15 de agosto, a ABH, em parceria com a BioDecision Analytics, apresentou o webinar Inteligência de Dados e Doença de Huntington: novas perspectivas terapêuticas. Nossos agradecimentos à Dra. Luciana Munhoz pela mediação do evento e ao Dr. Rodrigo Araldi pela exposição dos resultados da pesquisa para toda a comunidade de DH. ➡️ O […]

O post Em estudo inédito, pesquisadores brasileiros descobrem novos alvos terapêuticos para a doença de Huntington apareceu primeiro em ABH - Associação Brasil Huntington.

Česká SPHCH sa na nás obrátila s ponukou účasti na sledovacej štúdii pána docenta Klempířa. (špičkového odborníka na HCH.) Text e-mailu : Prosíme o pomoc se sledovací studií, která monitoruje kvalitu života pacientů s HCH a pečujících osob. Jedná se o několik dotazníků, které lze vyplnit elektronicky (v PDF) a zaslat zpět e-mailem, případně vytisknout, …

Sledovacia štúdia Čítajte viac »