News

News

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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Can Two Brothers Struck With ‘Lorenzo’s Oil’ Disease Be Saved?

One brother is living a normal life; the other isn’t. Their story illustrates the potential of gene therapy. Source: The Daily Beast | By: KAREN WEINTRAUB | 11.01.17 9:00 AM ET In the early months of 2014, Brandon Rojas was a typical 6-year-old: healthy with a sunny disposition, a love of sports,

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Viking Therapeutics Presents Results from Study of VK0214 in In Vivo Model of X-ALD

SAN DIEGO, Oct. 23, 2017 (PRNewswire) — Viking Therapeutics, Inc. (“Viking”) (NASDAQ: VKTX), a clinical-stage biopharmaceutical company focused on the development of novel therapies for metabolic and endocrine disorders, today announced positive results from a 25-week proof-of-concept study of VK0214 in an in vivo model of X-linked adrenoleukodystrophy (X-ALD).  The final study data were

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ALD Mom & Board Member Janis Sherwood Featured on NPR’s “All Things Considered” for Newborn Screening Advocacy

To listen to the radio program, click here. The following article was written on www.npr.org by Anna Gorman: Kerri De Nies received the news this spring from her son’s pediatrician: Her chubby-cheeked toddler has a rare brain disorder. She’d never heard of the disease — adrenoleukodystrophy, or ALD — but soon felt

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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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