Rare Disease Workshop: Challenges and barriers for gene therapies in rare neurological disorders: preparing the next generation of clinicians and researchers,

Pubblicato il 08/05/2023
ERN - Workshop
Istituto Scienze Neurologiche
  • Dove Barcelona, Spain
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Advances in gene therapies are completely changing the possibilities we used to have to deal with devastating rare neurological disorders and dramatically changing our expectations regarding outcomes in these patients. More than 50 gene therapies could be in the clinical ground in the next 10 years, but there are still lots of uncertainties and challenges we need to cope with.

The aim of this course is to discuss known barriers, challenges, and uncertainties in gene replacement therapies including the vision of different stakeholders (basic researchers, clinicians, patients, regulators, payers, and industry.