News

News

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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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 spearheading the effort. Their annual skateboarding fundraiser, EZ

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“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.

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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.

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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

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Bluebird Bio’s Gene Therapy Shows Promise for ALD Treatment

By: Ben Fidler April 20th, 2016 BOSTON – Cerebral adrenoleukodystrophy is a rare, crippling neurological disease that leaves its patients, typically boys, severely disabled and ultimately dead in a matter of years. Only painful, dangerous bone marrow transplants can stop the progression of CALD. But while the transplants are effective

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“Aidan’s Law” Petition for National ALD Newborn Screening Reaches 50,000 Signatures

Elisa Seeger (pictured above) and The Aidan Jack Seeger Foundation reached a milestone this week when their petition on Change.org received 50,000 signatures supporting “Aidan’s Law” (S. 2641 & H.R. 4692) to enact adrenoleukodystrophy (ALD) newborn screening nationwide. The bill would require newborn screening of ALD in all 50 US

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We’re Now Members of the National Organization for Rare Disorders (NORD)

It is with great excitement we announce that we are now members of the National Organization for Rare Disorders (NORD). Our goal is to increase the exposure of ALD, and to coordinate with other rare disease organizations to advocate for legislation and raise awareness of all rare diseases. NORD is a United States based 501(c)3 tax

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