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All Posts by The Myelin Project

Newborn Screening Family Education Survey

This survey is being conducted by Expecting Health, a non-profit organization focused on engaging families and health professionals in pregnancy and newborn health: https://www.surveymonkey.com/r/P5D363H. If you agree to participate, you will be presented with an online survey. The goal of this survey is to learn about families’ needs and priorities

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Orchard Therapeutics’ OTL-200 Receives Rare Pediatric Disease Designation from FDA for Treatment of Metachromatic Leukodystrophy

Orchard’s fourth Rare Pediatric Disease Designation for autologous ex vivo gene therapy. Boston, USA and London, UK, May 03, 2018 / B3C newswire / — Orchard Therapeutics, a leading commercial stage company dedicated to transforming the lives of patients with rare diseases through innovative gene therapies, today announced that the

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NORD Statement on Governor Bevin’s Veto of Kentucky Senate Bill 7

Washington, D.C., April 11, 2018 – The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, released the following statement regarding Governor Matt Bevin’s Veto of SB 7, the Kentucky Rare Disease Advisory Council: “Last week, Kentucky Governor Matt Bevin

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Magenta Therapeutics Announces First Patient Transplanted with MGTA-456 in Phase 2 Study in Inherited Metabolic Disorders

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Magenta Therapeutics, a biotechnology company developing novel medicines to bring the curative power of bone marrow transplant to more patients, today announced treatment of the first patient with an inherited metabolic disorder in a Phase 2 study of MGTA-456, an expanded cord blood stem cell product. MGTA-456 is

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Progress Report: Dr. Florian Eichler’s AMN Gene Therapy Pilot Study

Last year, The Myelin Project (in partnership with the Cure ALD Foundation) granted $50,000 to Dr. Florian Eichler’s pilot study of gene therapy in adrenomyeloneuropathy (AMN). Dr. Eichler and his team established an AMN animal model proof of concept showing a gene therapy approach could provide benefit where no treatment

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Making Miracles Possible for Kids with Leukodystrophy

In 2013, a year after Pat and Maria Carr learned that their youngest child had one of the 50 forms of leukodystrophy, a degenerative, genetic neurological condition, they knew they wanted to help. It was too late to save Cal, but the Carrs were determined to give children with leukodystrophy

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