Tagged as: adrenoleukodystrophy

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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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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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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bluebird bio to Present Clinical Data from Study of Lenti-D in ALD at CNS Annual Meeting

CAMBRIDGE, Mass.–(BUSINESS WIRE)–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 the initial cohort of 17 patients in the ongoing Phase 2/3 Starbeam Study (ALD-102) evaluating Lenti-D™ investigational

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September is Leukodystrophy Awareness Month

September is Leukodystrophy Awareness Month! Share your story on Facebook, Twitter, and Instagram using the hashtag #LeukodystrophyAwareness and help us spread the word to ensure that leukodystrophies become a household name. Leukodystrophies are a group of rare, progressive, metabolic, genetic diseases that affect the brain, spinal cord and often the peripheral nerves.

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NeuroVia Raises $14M to Test Drug for ALD

Frank Vinluan July 20th, 2017 @frankvinluan Xconomy Boston —   Patients who have the rare genetic disorder cerebral adrenoleukodystrophy (ALD) have few options to stave off the progressive decline of brain and muscle function. Within three to five years of diagnosis, the disease typically becomes fatal. A startup called NeuroVia has

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bluebird bio Announces Clinical Data from Study of Lenti-D™ Drug in ALD

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Jun. 26, 2017– bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, announced topline interim data from the initial cohort of 17 patients in the ongoing Phase 2/3 Starbeam Study (ALD-102) evaluating Lenti-D™ investigational

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In Loving Memory of an AMN Hero: Mark Bostock

The ALD/AMN community lost one of its most cherished heroes yesterday.  Mark Bostock was one in a million. Anyone who knew Mark, had the distinct pleasure of being acquainted with an inspirational, kind-hearted, and determined man who could easily put a smile on the faces of anyone that crossed his path. Mark Bostock

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Connecticut Becomes 2nd State to Implement ALD Newborn Screening

Since Jean and Dr. Jack Kelley’s son Brian was diagnosed with adrenoleukodystrophy at the age of six, the parents have advocated for a statewide screening program to test all newborns for the disorder in the hope of saving another boy’s life. Three years after a bill mandating ALD be added

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