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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 U.S. Food and

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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 vetoed

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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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Washington DC Introduces ALD Newborn Screening Legislation

Council member Brandon Todd of Washington DC introduced the “Newborn Screening Amendment Act of 2018,” which adds the test for Adrenoleukodystrophy, better known as ALD, to the list of newborn screenings that District hospitals must offer. ALD is a rare genetic brain disorder, affecting approximately one in every 18,000 people,

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We LOVE Miracle Flights… and Here’s Why

Miracle Flights is a nonprofit organization that helps sick children fly to distant medical care. Since 1985, Miracle Flights has established itself as the nation’s leading nonprofit health and welfare flight organization, providing financial assistance for flights so that those with serious illnesses may receive life-altering, life-saving medical treatment and

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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 is currently available.

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In Loving Memory of an AMN Hero: Wyatt Zmrzel

It is with great sadness that we announce the passing of Wyatt Zmrzel, son of our Board Member Diane Love, who was tragically killed in a car accident just days after Christmas. Wyatt had AMN and suffered from epileptic seizures but he never let either of those things hold him

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NORD Launches 7,000 Mile Rare Movement

Danbury, CT, Jan. 16, 2018— The National Organization for Rare Disorders (NORD)®, the leading nonprofit organization dedicated to helping the 30 million Americans with rare diseases, today announced its new 7,000 Mile Rare Movement, challenging Americans to pledge dollars to walk, run or bike 7,000 miles collectively throughout the month of February. The

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