Tagged as: adrenoleukodystrophy

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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Viking Therapeutics Announces Results of VK0214 in x-ALD Mouse Model

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 top-line results from a proof-of-concept study of VK0214 in a mouse model of X-linked adrenoleukodystrophy (X-ALD).  The results of this study showed that VK0214 rapidly

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