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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Dr. Ian Duncan’s Study Reveals New Clues into Myelin Disorder H-ABC

Dr. Ian Duncan, Chairman of The Myelin Project’s Scientific Advisory Board, has made a significant discovery into the genetic myelin disorder known as H-ABC. After a 29-year quest, Ian Duncan, a professor of veterinary medicine at the University of Wisconsin-Madison, has finally pinpointed the cause of a serious neurologic disease

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Minoryx Therapeutics Completes Phase 1 Clinical Trial of ALD/AMN Drug

22 March 2017 | By: BioSpectrum Minoryx Therapeutics, a drug development company specialized in the discovery of new drugs for orphan diseases, announced that it has successfully completed its phase 1 trial with MIN-102. MIN-102 targets X-linked adrenoleukodystrophy (X-ALD), a rare and chronically debilitating life threatening neurodegenerative disease. There are

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Minoryx Launches Phase 2/3 Trial for AMN Drug

Minoryx Therapeutics, the Spanish pharmaceutical company based in Barcelona and headed up by Mark Martinell, is preparing to launch Phase II/III trial for their drug MIN-102, which could potentially alleviate AMN symptoms. To support the effort, they have recruited ALD Life friends and renowned ALD/AMN specialists Professor Patrick Aubourg, Dr Florian Eichler,

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Today, President Obama signed into law the 21st Century Cures Act, a game-changing bill for medical innovation.  The bill includes many provisions that will improve the discovery, development, and delivery of orphan therapies for rare disease patients, including: An extension of the Rare Pediatric Disease Priority Review Voucher Program, which

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Viking Therapeutics Announces Positive Data from VK0214 Study in Mouse Model of X-ALD

“Viking Therapeutics announced the presentation of positive data from a proof-of-concept study of VK0214 in a mouse model of X-linked adrenoleukodystrophy (X-ALD).  The study successfully achieved its primary objective, which was to demonstrate the ability of VK0214 to lower plasma very long chain fatty acid (VLCFA) levels after six weeks

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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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