The Portuguese Association for CDG (APCDG) has developed 2 short surveys to assess the needs and solutions for future therapies among CDG Families and Professionals.
Following on from the first webinar on the transition from childhood to adulthood, Work Package 7, in collaboration with the SSIEM, has developed a second webinar on the transition from childhood to adulthood in Inherited Metabolic Disorders.
The 2nd Course on Low Protein diet for Metabolic Patients will took place on 29th March 2019 at Hospital 12 de Octubre, in Madrid. El 29 de Marzo de 2019 tendrá lugar en el Hospital 12 de Octubre, en Madrid, el Segundo Taller de Dieta Hipoproteica para Pacientes Metabólicos.
To improve the realisation of clinical trials within the network, and adequately adapt the protocols to patients' expectations, MetabERN wishes to understand your perception of clinical trials.
Building awareness of rare diseases is so important because 1 in 20 people will live with a rare disease at some point Rare Disease Day in their life.
ERN Clinical Patient Management System Webinar to introduce CPMS 17.0 to be held for all ERN Members. CPMS 17.0 will be released at the beginning of March.
This 15th annual symposium is designed for basic, translational and clinical researchers, patient advocacy groups, clinicians, and all others who are interested in learning more about the latest discoveries related to lysosomal diseases and the clinical investigation of these advances.
3rd International Homocystinurias Patient-Expert Meeting on Thursday 28th February – Friday 1st March 2019 in Rome, Italy.
RARE2019 will follow on from the success of its predecessor, addressing the challenges of Rare Diseases Treatments as well as new therapies on the horizon. It will expand on so-called "generic topics", such as clinical trials' design, funding for research, digital health, big data on the one hand and focusing on the individual patients for innovation in both drug development and management.
Annual MetabERN Board meeting starting on Thursday 4 April (start time 14.00) to Saturday 6 April (finishing at 13.00).