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Tagged as: adrenoleukodystophy

Can Two Brothers Struck With ‘Lorenzo’s Oil’ Disease Be Saved?

One brother is living a normal life; the other isn’t. Their story illustrates the potential of gene therapy. Source: The Daily Beast | By: KAREN WEINTRAUB | 11.01.17 9:00 AM ET In the early months of 2014, Brandon Rojas was a typical 6-year-old: healthy with a sunny disposition, a love

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Viking Therapeutics Presents Results from Study of VK0214 in In Vivo Model of X-ALD

SAN DIEGO, Oct. 23, 2017 (PRNewswire) — 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 results from a 25-week proof-of-concept study of VK0214 in an in vivo model of X-linked adrenoleukodystrophy (X-ALD). The

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newborn-screening

ALD Mom & Board Member Janis Sherwood Featured on NPR’s “All Things Considered” for Newborn Screening Advocacy

To listen to the radio program, click here. The following article was written on www.npr.org by Anna Gorman: Kerri De Nies received the news this spring from her son’s pediatrician: Her chubby-cheeked toddler has a rare brain disorder. She’d never heard of the disease — adrenoleukodystrophy, or ALD — but

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bluebird bio Publishes Interim Data from Starbeam Study of Lenti-DTM Drug in Cerebral Adrenoleukodystrophy (CALD) Patients

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Oct. 4, 2017– bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for serious genetic diseases and T cell-based immunotherapies for cancer, today announced that interim data from an initial cohort of 17 patients in the ongoing Phase 2/3 Starbeam Study (ALD-102)

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model-mouse-x-linked

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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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

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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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Newborn Screening

“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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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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