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Orchard Therapeutics’ gene therapy for metachromatic leukodystrophy (MLD) resulted in motor ability improvements more than six times over the target endpoint, according to data released at a recent symposium.
Article Link to be found here : https://www.prnewswire.com/news-releases/pmd-foundation-announces-international-natural-history-study-on-individuals-with-pelizaeus-merzbacher-disease-300911949.html
Orchard Therapeutics has said it plans to file for approval for a gene therapy for the life-threatening rare disease metachromatic leukodystrophy (MLD) in Europe, after publishing new data
On 23 July 2019 Genetic Alliance shared their patient charter on newborn screening with the All Party Parliamentary Group on Rare, Genetic and Undiagnosed Conditions.
View our July newsletter – We’ve had plenty to shout about since our re-launch.
The BIMDG psychologists group work with individuals and families affected by inherited metabolic disorders (IMD).
