News

News

“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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The Leukodystrophy Care Network (LCN) – Establishing Care Guidelines

Imagine your child experiencing an unexplained sudden loss of basic functions – a toddler who once constantly walked and ran suddenly stumbling with each step; an infant with a healthy appetite who once smiled and cooed now crying inconsolably for every waking hour and only eating a few ounces of

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Forbes Health Writer, Rita Rubin, on ‪Lorenzo’s Oil‬, the Odones, & ‪‎ALD‬ ‪Newborn Screening

Lorenzo’s Oil Could Not Cure Lorenzo, But Newborn Screening Is Expected To Save Others From His Fate By: Rita Rubin Forbes.com Lorenzo’s Oil, a 1992 film starring Nick Nolte and Susan Sarandon, depicted Augusto and Michaela Odone’s quest for a treatment that could save their son Lorenzo from dying of

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Legislation Introduced to Enact Nationwide Newborn Screening for ALD

The Aidan Jack Seeger Foundation is excited to announce a new initiative to expand newborn screening for ALD nationally. With the introduction of S.2641 by the Senator Charles Schumer of New York and companion Bill H.R. 4692 by Congresswoman Yvette Clarke of New York, the Foundation is working to have every state test for ALD in

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Meet Justin D’Agostino: Our first AMN Patient Liaison

Justin D’Agostino was diagnosed with AMN in 2007. His younger brother was diagnosed a year earlier and passed away in 2010. Recently, Justin has endeavored to become active in the ALD community. He is passionate about disease management and quality of life concerns. Justin currently resides in Phoenix, Arizona. He enjoys

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