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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NEWS
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
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
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
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
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
“Women with ALD” Webinar By Dr. Marc Engelen
Dr. Marc Engelen, Pediatric Neurologist at the Academic Medical Center of Amsterdam, gave a webinar presentation last month titled, “Women with ALD.” This was the 11th installment of ALD Connect’s Webinar Series. To see more webinars from ALD Connect, click here. For more information, visit www.aldconnect.org.
Washington State Closer to ALD Newborn Screening
The Washington State Department of Health voted unanimously in favor of adding ALD to Washington state’s newborn screening program following an advisory committee meeting that took place in January of this year. The Ethan Zakes Foundation, created by Brad and Nancy Zakes in memory of their son, Ethan, has been
Minoryx Therapeutics initiates phase 1 clinical trial of MIN-102
Minoryx Therapeutics, a drug development company specialized in the discovery and development of new drugs for orphan diseases, announced the initiation of its first-in-man Phase 1 clinical trial for its lead compound MIN-102. MIN-102 is a differentiated PPAR gamma agonist with a superior profile for central nervous system related diseases.
Minnesota Department of Health Adds ALD to Newborn Screening Program
The following is a press release from The Minnesota Department of Health: “The Minnesota Department of Health (MDH) today announced it has added three disorders – Mucopolysaccaridosis Type 1 (MPS-1), Pompe Disease and X-linked Adrenoleukodystrophy (X-ALD) – to its list of conditions for which all newborns in the state are