News

News

Meet the CEO of Minoryx, the Spanish Biotech Fighting Rare Disease

By: Clara Rodríguez Fernández 10/07/2017 Having previously worked in Crystax Pharmaceuticals and Oryzon Genomics, Marc Martinell is now CEO of one of Spain’s most successful biotechs. Minoryx Therapeutics is developing what could be the first effective treatment for X-linked adrenoleukodystrophy (X-ALD), a rare disorder of the central nervous system (CNS). After a successful Phase I trial completed earlier

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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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National Organization for Rare Disorders (NORD) Vows to Resist the American Healthcare Act (AHCA)

The following is a message from the National Organization for Rare Disorders (NORD) regarding the repeal of the Affordable Care Act (ACA) and the recent passing of the American Health Care Act (AHCA): “Today the House of Representatives passed American Health Care Act (AHCA). We are very disappointed the House

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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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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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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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Small molecule helps fix ‘Lorenzo’s Oil’ gene mutation

Sobetirome partially makes up for lost protein function By: Stu Borman Researchers have identified a small organic molecule that may help fix the genetic defect that causes X-linked adrenoleukodystrophy (X-ALD), the rare disease featured in the 1992 movie “Lorenzo’s Oil.” X-ALD, which affects 1 in 20,000 to 50,000 people worldwide, typically

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