Tagged as: x-ALD

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 of sports,

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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 final study data were

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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 soon felt

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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) evaluating Lenti-D™

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bluebird bio to Present Clinical Data from Study of Lenti-D in ALD at CNS Annual Meeting

CAMBRIDGE, Mass.–(BUSINESS WIRE)–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 the initial cohort of 17 patients in the ongoing Phase 2/3 Starbeam Study (ALD-102) evaluating Lenti-D™ investigational

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September is Leukodystrophy Awareness Month

September is Leukodystrophy Awareness Month! Share your story on Facebook, Twitter, and Instagram using the hashtag #LeukodystrophyAwareness and help us spread the word to ensure that leukodystrophies become a household name. Leukodystrophies are a group of rare, progressive, metabolic, genetic diseases that affect the brain, spinal cord and often the peripheral nerves.

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Minoryx Therapeutics Completes Phase 1 Clinical Trial of ALD/AMN Drug

22 March 2017 | By: BioSpectrum Minoryx Therapeutics, a drug development company specialized in the discovery of new drugs for orphan diseases, announced that it has successfully completed its phase 1 trial with MIN-102. MIN-102 targets X-linked adrenoleukodystrophy (X-ALD), a rare and chronically debilitating life threatening neurodegenerative disease. There are

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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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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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