News

News

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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EveryLife Foundation’s NBS Initiative in Florida

Last week, the Everyday Life Foundation presented to Florida’s Genetics and Newborn Screening Advisory Council on their efforts to advance early diagnosis and treatment through robust newborn screening programs across the United States. Currently, Florida screens for every disease recommended by experts except for MPS I, Pompe, and X-ALD. The Foundation will work

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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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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

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Giving Tuesday

We have a day for giving thanks. We have two for getting deals. Now, we have #GivingTuesday, a global day dedicated to giving back. Today, charities, families, businesses, community centers, and students around the world are coming together for one common purpose: to celebrate generosity and to give. This #Giving

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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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ALD Newborn Screening Update in California

We are excited to announce that we received the following message from the California Department of Public Health (CDPH) yesterday: “We are pleased to announce that starting September 21, 2016 the #California #NewbornScreening (#NBS) Program will be expanding to include routine screening for #Adrenoleukodystrophy (#ALD). State Assembly Bill 1559 mandated that the California

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