Research

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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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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Mollii Suit: Assistive Device for Muscle Stiffness, Spasticity, & Motor Disability

The Mollii Suit is a new assistive device developed by Inerventions, a Swedish company, that can “relax spastic, tense, and aching muscles.”  The company was started by Mollii inventor Fredrik Lundqvist in 2009. According to their website, Mollii is currently being used by people with cerebral palsy, stroke, spinal cord injury,

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Researchers at the University of Buffalo Study Novel Proteins’ Role in Myelination

M. Laura Feltri, MD, professor of biochemistry and neurology, is leading research to determine whether a new family of molecules prevents demyelination and nerve degeneration in patients with peripheral nerve diseases. Research Builds on 2014 Finding The novel molecules, called prohibitins, are required for nerves to form correctly and remain healthy.  Feltri and

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The Myelin Project & Cure ALD Foundation Grant $50,000 to Dr. Florian Eichler’s AMN Gene Therapy Study

The Myelin Project in partnership with the Cure ALD Foundation, has granted $50,000 to Dr. Florian Eichler’s pilot study of gene therapy in adrenomyeloneuropathy (AMN). Dr. Eichler and his team have established an AMN animal model proof of concept showing a gene therapy approach could provide benefit where no treatment is currently

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Researchers receive $1.9 million NIH grant to better understand leukodystrophies

A team of Wayne State University researchers recently received a $1.9 million grant from the National Eye Institute of the National Institutes of Health to better understand leukodystrophies (LD) and genetic Leukoencephalopathies (gLE), rare genetic disorders affecting the white matter — myelin — in the central nervous system. Patients diagnosed

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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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Meet the CEO of Minoryx, the Spanish Biotech Fighting Rare Disease

By: Clara Rodríguez Fernández 10/07/2017 Having previously worked in Crystax Pharmaceuticals and Oryzon Genomics, Marc Martinell is now CEO of one of Spain’s most successful biotechs. Minoryx Therapeutics is developing what could be the first effective treatment for X-linked adrenoleukodystrophy (X-ALD), a rare disorder of the central nervous system (CNS). After a successful Phase I trial completed earlier

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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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