Charlie’s Head Shave for Alex, The Leukodystrophy Charity
Charlie has decided to shave his head to support his Son, George
Here are the latest news stories from Alex TLC and about topics we think you'll find useful.
Charlie has decided to shave his head to support his Son, George
Metabolic Support UK Survey Metabolic Support UK have done some incredible work into the important issue of families struggling to obtain travel insurance to provide coverage for their inherited metabolic disorder. The survey results are available to view and actions MSUK have taken to help.
Genomics England Survey Which conditions should be screened for for as part of the Newborn Genomes Programme? Have your say by completing Genomics England survey by Tuesday 14th June💬 The survey is to test the wording of some of the draft principles that will guide Genomics England in choosing the conditions to screen for in the newborn babies’ genomes. To find out more❓: Genomics England Website To access the survey 📝 : Newborn Genomes Programme Survey
Leukodystrophy and Motherhood One of our beneficiaries has written a wonderful and powerful book of poems about leukodystrophy and motherhood. She wants to share it with other families affected by rare disease to help others by letting them know they aren’t alone.
Gene Therapy for AMN Clinical Trial Update SwanBio Therapeutics have announced additional preclinical data that supports Clinical Advancement of First AAV-Based Gene Therapy for Adrenomyeloneuropathy. SBT101 preclinical studies demonstrate improvement in behavioral and biochemical markers of disease. Further support for dosing strategy for Phase 1/2 study initiating later in 2022.
Scientific Symposium & Family Conference United Leukodystrophy are holding their Scientific Symposium & Family Conference at the end of June.