This working group represents a collaborative initiative uniting three European Reference Networks: EURO-NMD (lead), ERN-RND and EpiCARE.
The working group will address the following topics:
– Current practices regarding genetic therapies in neurological disorders in Europe
– Regulatory and health care cost-related challenges common to all ERNs
– Role of ERNs in gene therapy development, clinical trials, and policy around marketed treatments in Europe.
More specifically, the focus will be on exchanging ideas for actions addressing aspects of gene therapy that are transversal to the 3 ERNs:
– Approved therapies and related practices
– Therapies currently in clinical trials
– Access to gene therapies
– Orphan gene therapies
