18 – 19 November | First International conference on Rare Diseases and Paediatric Research Continue reading “18 – 19 November | First International conference on Rare Diseases and Paediatric Research”
18 – 19 November | MDS 1st Movement Disorders Clinical Practice Conference Continue reading “18 – 19 November | MDS 1st Movement Disorders Clinical Practice Conference”
4 November | Drug repurposing for rare diseases workshop series Continue reading “4 November | Drug repurposing for rare diseases workshop series”
Editorial: Networks in Movement Disorders. To Move or Not to Move Continue reading “Editorial: Networks in Movement Disorders. To Move or Not to Move”
25 – 27 October | Training: Pluripotent stem cells for rare disease research: banking, data, application (registration deadline 27 September) Continue reading “25 – 27 October | Training: Pluripotent stem cells for rare disease research: banking, data, application (registration deadline 27 September)”
Publication: “Preparing n-of-1 Antisense Oligonucleotide Treatments for Rare Neurological Diseases in Europe: Genetic, Regulatory, and Ethical Perspectives” Continue reading “Publication: “Preparing n-of-1 Antisense Oligonucleotide Treatments for Rare Neurological Diseases in Europe: Genetic, Regulatory, and Ethical Perspectives””
15-16 October 2021 | Italian Mitochondrial Disease Conference 2021 Continue reading “15-16 October 2021 | Italian Mitochondrial Disease Conference 2021”
15 & 22 October | MDS-ES and EHDN Joint Online Course Series: Huntington’s Disease: From Foundational Principles to Assessment and Treatment Continue reading “15 & 22 October | MDS-ES and EHDN Joint Online Course Series: Huntington’s Disease: From Foundational Principles to Assessment and Treatment”
9 November 2021 | ERN-RND webinar “Clinical Outcome Assessments in Ataxias” Continue reading “9 November 2021 | ERN-RND webinar “Clinical Outcome Assessments in Ataxias””
12 October 2021 | ERN-RND webinar “Functional gait disorders: a sign-based approach” Continue reading “12 October 2021 | ERN-RND webinar “Functional gait disorders: a sign-based approach””
19 October 2021 | ERN-RND webinar “Oligonucleotide therapies: a new class of drugs that allow precise genetic targeting” Continue reading “19 October 2021 | ERN-RND webinar “Oligonucleotide therapies: a new class of drugs that allow precise genetic targeting””
18 November 2021 | ERN-RND webinar “Pure autonomic failure: really that pure?” Continue reading “18 November 2021 | ERN-RND webinar “Pure autonomic failure: really that pure?””
26 October 2021 | ERN-RND webinar “Pelizaeus-Merzbacher disease and related disorders” Continue reading “26 October 2021 | ERN-RND webinar “Pelizaeus-Merzbacher disease and related disorders””
7 October 2021 | ERN-RND webinar “x-linked adrenoleukodystrophies-Update” Continue reading “7 October 2021 | ERN-RND webinar “x-linked adrenoleukodystrophies-Update””
3-7 October 2021 | XXV World Congress of Neurology Continue reading “3-7 October 2021 | XXV World Congress of Neurology”
Available from EJP RD: MOOC Diagnosing Rare Diseases: from the Clinic to Research and back Continue reading “Available from EJP RD: MOOC Diagnosing Rare Diseases: from the Clinic to Research and back”
September 2021 | Dystonia Awareness Month Continue reading “September 2021 | Dystonia Awareness Month”
28 September 2021 | ERN-RND webinar “Treatable dystonias & dystonia in inborn errors of metabolism” Continue reading “28 September 2021 | ERN-RND webinar “Treatable dystonias & dystonia in inborn errors of metabolism””
17-22 September 2021 | MDS Virtual Congress 2021 Continue reading “17-22 September 2021 | MDS Virtual Congress 2021”
14 September 2021 | ERN-RND webinar “Genetic forms of Parkinson’s disease” Continue reading “14 September 2021 | ERN-RND webinar “Genetic forms of Parkinson’s disease””
9-11 September 2021 | EHDN plenary meeting Continue reading “9-11 September 2021 | EHDN plenary meeting”
New EJP RD training: “Pluripotent stem cells for rare disease research: banking, data, application” Continue reading “New EJP RD training: “Pluripotent stem cells for rare disease research: banking, data, application””
Launch of the ERN Research Training Workshops funding opportunity Continue reading “Launch of the ERN Research Training Workshops funding opportunity”
4 September 2021 | Dystonia Europe Annual Conference – Dystonia Day Continue reading “4 September 2021 | Dystonia Europe Annual Conference – Dystonia Day”
Review article: “Evolving concepts in progressive supranuclear palsy and other 4-repeat tauopathies” Continue reading “Review article: “Evolving concepts in progressive supranuclear palsy and other 4-repeat tauopathies””
Research article: “Biallelic PI4KA variants cause neurological, intestinal and immunological disease” Continue reading “Research article: “Biallelic PI4KA variants cause neurological, intestinal and immunological disease””
Research article: “Neurofilament light chain and glial fibrillary acidic protein levels in metachromatic leukodystrophy” Continue reading “Research article: “Neurofilament light chain and glial fibrillary acidic protein levels in metachromatic leukodystrophy””
New interview available – meet Nicole Wolf from Emma Children’s Hospital/Amsterdam University Medical Centers, the Netherlands Continue reading “New interview available – meet Nicole Wolf from Emma Children’s Hospital/Amsterdam University Medical Centers, the Netherlands”