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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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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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Minoryx Therapeutics announces dosing of first patient in phase 2/3 clinical study of MIN-102 in patients with AMN

ADVANCE trial expected to initiate patient recruitment shortly in several other EU countries, followed by the US MATARO, Spain I January 4, 2018 I Minoryx Therapeutics, a company specialized in the development of new drugs for orphan diseases, today announces the initiation of treatment of the first two patients in the ADVANCE

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Cambridge Researcher Wins 2017 Barancik Prize for Pioneering Work on Myelin Repair

A University of Cambridge researcher, Robin Franklin, has been awarded the 2017 Barancik Prize for Innovation in MS Research for his work on myelin repair and as a potential way of treating multiple sclerosis (MS). Franklin is a senior scientist at the Wellcome Trust-MRC Cambridge Stem Cell Institute and director of the Cambridge MS Society Centre for Myelin Repair. He

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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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bluebird bio Publishes Interim Data from Starbeam Study of Lenti-DTM Drug in Cerebral Adrenoleukodystrophy (CALD) Patients

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Oct. 4, 2017– bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for serious genetic diseases and T cell-based immunotherapies for cancer, today announced that interim data from an initial cohort of 17 patients in the ongoing Phase 2/3 Starbeam Study (ALD-102) evaluating Lenti-D™

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