Family Perspectives on Newborn Screening for X-Linked ALD in California
An interesting paper exploring the experiences of how a diagnosis of ALD is communicated to parents through the Californian newborn screening programme.
Here are the latest news stories from Alex TLC and about topics we think you'll find useful.
An interesting paper exploring the experiences of how a diagnosis of ALD is communicated to parents through the Californian newborn screening programme.
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
View our July newsletter – We’ve had plenty to shout about since our re-launch.
On 15th May 2019, ALD relaunched as Alex, The Leukodystrophy Charity!