Disease Group: Leukoencephalopathy
Genetic therapies and therapy developments for rare movement disorders
State of the art of clinical stage gene therapies for Leukodystrophies
Newborn screening in leukodystrophies
Advanced therapies and clinical trials in Leukodystrophies
Haematopoietic Stem Cell Transplantation in Leukodystrophies
Telerehabilitation in RND: an update
Krabbe disease – natural history and treatment options
Vanishing white matter
13 Sep. 2022
Speaker: Marjo van der Knaap
VU University Medical Center Amsterdam, Netherlands
Importance of autopsies in leukodystrophies
Measuring disease severity in chronic progressive myelopathy
Pelizaeus-Merzbacher disease and related disorders (pediatric focus)
26 Oct. 2021
Speaker: Nicole Wolf
VU University Medical Center Amsterdam, Netherlands;
Emma Children’s Hospital, Amsterdam, Netherlands
Oligonucleotide therapies: a new class of drugs that allow precise genetic targeting
Functional gait disorders: a sign-based approach
X-Linked leukodystrophies – Update
Adult leukodystrophies/early symptoms of late-onset Leukodystrophies
Metachromatic Leukodystrophy – update
15 Jun. 2021
Speaker: Samuel Gröschel & Ingeborg Krägeloh
University Hospital Tübingen, Germany
MRI pattern recognition in leukodystrophies
8 Jun. 2021
Speaker: Nicole Wolf
VU University Medical Center Amsterdam, Netherlands;
Emma Children’s Hospital, Amsterdam, Netherlands
Gene therapy in neuromuscular and mitochondrial disorders
Functional movement disorders: a diagnostic guide
Treatment of spasticity in HSP and leukodystrophies
6 Oct. 2020
Speaker: Annemieke Buizer & Laura Bonouvrié
VU University Medical Center Amsterdam, Netherlands
How can we develop and implement evidence based rehabilitation in rare disorders?
29 Sep. 2020
Speaker: Hortensia Gimeno
Guy’s and St Thomas’ NHS Foundation Trust, London, United Kingdom
How to assess and manage spastic gait in rare diseases
Goal setting and outcome measures of interventions in pediatric rehabilitation.
18 Jun. 2020
Speaker: Annemieke Buizer
VU University Medical Center Amsterdam, Netherlands;
Emma Children’s Hospital, Amsterdam, Netherlands