Organización destacada: Red de Enfermedades Raras Miastenia Gravis (MGNet)
The Myasthenia Gravis Rare Disease Network (MGNet) is a consortium of academic medical centers partnering with the Myasthenia Gravis Foundation of America and Conquer MG as well as collaborators in other research groups and industry. We are working together to enhance therapeutic development for this rare disease.
MGNet is part of the National Institutes of Health’s Rare Diseases Clinical Research Network (RDCRN).
Myasthenia gravis (MG) is an autoimmune disorder caused by antibodies against proteins at the nerve-muscle communication point. Within that common, well-defined pathology, distinct subtypes of MG exist. The underlying cause and response to treatments likely vary based on patient age (early vs late onset), thymus abnormality (thymoma, thymic hyperplasia, or thymic atrophy), and antibody status (acetylcholine receptor antibody, muscle-specific kinase antibody, or an absence of detectable antibodies antibodies-seronegative MG). The causes of each subtype likely differ.
Existen terapias, pero tienen importantes efectos secundarios y algunos pacientes responden mal. Los estudios demuestran que, incluso con tratamiento, algunos pacientes tienen una mala calidad de vida.
El objetivo de MGNet es avanzar en el desarrollo terapéutico. Para ello tenemos cuatro objetivos específicos:
Mejorar las herramientas que tienen los científicos y los médicos para realizar ensayos clínicos mediante un seguimiento riguroso de los pacientes a lo largo del tiempo y evaluar mejores medidas de la evolución de los pacientes.
Identify objective measures (biomarkers) to show how the disease is responding to treatment and better predict treatment response or side effects, to improve the design of clinical trials and care of patients in clinical practice.
Enhance the education of young investigators focusing their careers on rare diseases and specifically MG.
Improve the awareness of scientists, physicians, and the public regarding the unique needs of patients with MG through the training of clinician-scientists and engagement of the patient and scientific communities in clinical research and therapeutic development.
MGNet está financiado por el Instituto Nacional de Trastornos Neurológicos y Accidentes Cerebrovasculares y el Centro Nacional para el Avance de las Ciencias Translacionales.
Enfermedades estudiadas - Miastenia Gravis (MG) y sus subtipos:
MG positiva a los anticuerpos del receptor de acetilcolina (AChR) de aparición temprana, que afecta principalmente a las mujeres
MG de inicio tardío de AChR Ab positivo con un sesgo de la enfermedad hacia los hombres
MG paraneoplásica asociada al timoma
Muscle-specific kinase (MuSK) Ab positive
AChR/MuSK antibody-negative MG.
For more information, visit https://rdcrn.org/mgnet.
MGNet ofrece numerosos vídeos educativos para dar a conocer la investigación clínica.
