NEWS

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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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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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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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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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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California A.L.D. Newborn Screening Program to Begin mid-September

We received word from the California Department of Public Health’s Genetic Disease Screening Program (GDSP) that adrenoleukodystrophy (ALD) screening will begin in mid-September. In 2013, The Myelin Project sponsored California Assembly Bill 1559 – authored by former Assemblyman, now California Senator Dr. Richard Pan – in order to mandate newborn screening of adrenoleukodystrophy (ALD) in California. On September 25th, 2014, Governor Jerry

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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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