News

News

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

Read More

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.

Read More

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,

Read More

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

Read More

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

Read More

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

Read More

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

Read More