Tagged as: ALD

NeuroVia Raises $14M to Test Drug for ALD

Frank Vinluan July 20th, 2017 @frankvinluan Xconomy Boston —   Patients who have the rare genetic disorder cerebral adrenoleukodystrophy (ALD) have few options to stave off the progressive decline of brain and muscle function. Within three to five years of diagnosis, the disease typically becomes fatal. A startup called NeuroVia has

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bluebird bio Announces Clinical Data from Study of Lenti-D™ Drug in ALD

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Jun. 26, 2017– bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, announced topline interim data from the initial cohort of 17 patients in the ongoing Phase 2/3 Starbeam Study (ALD-102) evaluating Lenti-D™ investigational

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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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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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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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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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bluebird bio to Present Clinical Data on Lenti-D in ALD at AAN 2016 Annual Meeting

CAMBRIDGE, Mass. – bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, today announced that interim data from the ongoing Phase 2/3 Starbeam Study (ALD-102) for the treatment of cerebral adrenoleukodystrophy (CALD) will be

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Rare Disease Day 2016

On February 29th, 2016, we attended Rare Disease Day at the State Capitol building in Sacramento, CA with other rare disease advocates to raise awareness of rare diseases and advocate for legislation by targeting the general public, policy makers, public authorities, industry representatives, researchers, and health professionals. In partnership with the National Organization for Rare

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ALD added to Recommended Uniform Screening Panel

Washington, D.C. – February 16th, 2016 – Secretary of the Department of Health & Human Services, Sylvia Burwell, approved the addition of adrenoleukodystrophy (ALD) to the Recommended Uniform Screening Panel (RUSP), the federal list of genetic diseases recommended for state newborn screening programs. The addition of ALD to the RUSP may expedite

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