Pacientes
Registro Internacional de Pacientes
Invitación a formar parte del Registro Internacional de Pacientes con ARSACS si usted o un familiar fue diagnosticado con ARSACS. Esta invitación es válida, incluso si ya se unió a otro registro de pacientes.
El Registro Internacional de Pacientes con ARSACS es un conjunto de información básica sobre pacientes diagnosticados con ARSACS, más allá del país de residencia. Su participación en este registro ayudará enormemente al desarrollo de tratamientos para este desorden. El objetivo del registro es facilitar el contacto entre los investigadores que trabajan y los pacientes que desean recibir información sobre la participación en ensayos clínicos. Además de ser una herramienta esencial para los investigadores, el registro de pacientes brinda una estimación del tamaño del mercado y del potencial para la inscripción de ensayos. Estos son aspectos fundamentales al momento de analizar las posibilidades de desarrollar medicamentos con las empresas farmacéuticas.
La comunidad investigadora necesita de los pacientes, y esta es una forma de que ayuden a encontrar un tratamiento.
ARSACS International Patient Registry
Registre international des patients ARSACS
Registro internazionale dei pazienti ARSACS
You are not alone
If you have been diagnosed with ARSACS, you are not alone. Persons affected with ARSACS are found all over the word. According to the Foundation’s ARSACS International Patient Registry statistics, ARSACS is found in all continents.
Your voice is very important. Make your voice heard by participating in ARSACS research.
PROSPAX
The Ataxia Charlevoix-Saguenay Foundation is one of the 3 patient advocacy organisations participating in the PROgression chart of SPAstic ataXias (PROSPAX) research project. PROPSPAX, a 3 year project launched in September 2020, is a novel collaborative effort between several neurologists across Canada and Europe.
It is an unique opportunity for patients to have their voice heard by researchers, to make sure that the questions they are trying to answer are the ones that are important to a person living with the condition. The PROSPAX project will focus on two conditions – ARSACS and SPG7. There is a patient survey currently under development and your participation in the survey is crucial. Stay tuned.
TREAT- ARCA
The Foundation is pleased to represent the ARSACS patients on the research project “Designing a toolbox of paradigmatic treatments for a targeted molecular medicine approach to autosomal-recessive ataxias”. The project has received funding from the European Union’s Horizon 2020 research and innovation programme under the European Joint Program Rare Disease.
The specific objective is the pre-clinical-research to develop effective therapies for rare diseases. It aims at designing and testing new complementary treatment strategies, including both repurposed and newly identified molecules, as well as gene therapies in two exemplary multisystemic flagship ARCAS: Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay (ARSACS) and COQ8A-ataxia (ARCA2). It is a 3 year project.
Preclinical and clinical trials
The Foundation is ready to conduct preclinical and clinical trials. It has the expertise and the tools available and is always looking at opportunities to do clinical trials. However, the participation of ARSACS patients is crucial to conduct such trials.
Research that heals so there is hope
The Foundation’s mission is to discover and develop a treatment for the Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay (ARSACS).
Progress has been made over the years in identifying the source of the disorder. Your support is crucial to continue funding high potential projects as well as a clinical trial.