Research

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

Read More

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

Read More

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,

Read More

PRESIDENT SIGNS 21st CENTURY CURES MEDICAL INNOVATION BILL INTO LAW

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

Read More

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

Read More

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

Read More