Neuromuscular Disorder News and Research

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TACT believes that Halo's HT-100 may effectively treat DMD

Halo Therapeutics, LLC, a clinical-stage biopharmaceutical company developing novel therapeutics for rare fibrotic diseases, announced today that the TREAT-NMD Advisory Committee on Therapeutics (TACT) has reviewed HT-100, Halo's drug candidate for Duchenne muscular dystrophy (DMD), and believes the compound has potential based on the proposed mechanism.

21 Mar 2012

PTC, Roche announce licensing agreement for SMA programme

Roche, PTC Therapeutics, Inc. and the SMA Foundation, announced today a licensing agreement for PTC's Spinal Muscular Atrophy (SMA) programme.

30 Nov 2011

Researchers to begin drug development projects for rare and neglected diseases

Researchers will begin drug development projects for rare and neglected diseases that include potential treatments for a musculoskeletal disorder, a cognitive dysfunction disorder, a virus that affects the central nervous system of newborns, a parasitic worm infection, a form of muscular dystrophy and a rare lung disease.

16 Nov 2011

Reduced levels of VAPB protein may play central role in causing ALS

An international team of scientists led by researchers at the University of California, San Diego School of Medicine have used induced pluripotent stem cells (iPSCs) derived from patients with amyotrophic lateral sclerosis (ALS) to reveal for the first time how reduced levels of a specific protein may play a central role in causing at least one inherited form of the disease.

22 Jun 2011

RRD enters partnership with NIH Therapeutics for Rare and Neglected Diseases program

RRD International, a strategy-driven biotech and pharmaceutical product development company, announced today that it has entered into a partnership with the National Institutes of Health (NIH) Therapeutics for Rare and Neglected Diseases (TRND) program as a regulatory support and development partner.

25 Jan 2011

Research finds alteration in KBTBD13 protein causes nemaline myopathy

Researchers from the Department of Anesthesiology, Uniformed Services University of the Health Sciences (USU), along with research teams from the National Institutes of Health and from Australia, the Netherlands and Spain, have identified a novel gene on chromosome 15q that, when altered, causes nemaline myopathy with cores, a rare inherited muscle disorder. The gene encodes a member of the BTB/Kelch family of proteins.

30 Nov 2010

2010 Mid-Atlantic Bio a great kick-off for hosting 2011 BIO International Convention

The 2010 Mid-Atlantic Bio today completed another successful conference hosting 728 registrants from 22 states and 4 countries, with more than 400 companies and 50 qualified investors participating. Dedicated to promoting the biotechnology industry in the Mid-Atlantic region, the conference is hosted collectively by the region's most influential bioscience and investor associations, the Mid-Atlantic Venture Association (MAVA), the Tech Council of Maryland/MdBio (MdBio) and the Virginia Biotechnology Association (VaBIO).

30 Oct 2010

ISIS-FXIRx and ISIS-SMNRx included in Isis Pharmaceuticals' development pipeline

Isis Pharmaceuticals, Inc. announced today that it has added two new drugs to its development pipeline, ISIS-FXIRx and ISIS-SMNRx.

4 Dec 2009

UT physicians treat Iraqi girl with myasthenia gravis

Plagued by a growing weakness that left her unable to walk, talk and even take a normal breath, 11-year-old Iraqi Aram Ali was a shell of the bright little girl she used to be.

16 Nov 2009

Three genes linked to Lou Gehrig's disease

Michigan Technological University researchers have linked three genes to the most common type of amyotrophic lateral sclerosis (ALS), generally known as Lou Gehrig's disease.

10 Sep 2009

Safeway raises funds to support neuromuscular disease medical research and therapies

Safeway Inc. announced today it has raised a record $10.2 million to support medical research and services for the millions of people living with neuromuscular diseases. The funds were raised during Safeway’s annual Muscular Dystrophy Association (MDA) campaign, a five-week endeavor to highlight the need to find cures and treatments for muscular dystrophy and related diseases, and presented during the Jerry Lewis MDA Telethon in Las Vegas Labor Day weekend.

9 Sep 2009

Muscular Dystrophy Canada to host the 44th Jerry Lewis Labour Day Telethon

Muscular Dystrophy Canada is hosting the 44th Annual Jerry Lewis Labour Day Telethon to support those living with a neuromuscular disorder.

7 Sep 2009

Protein from tick saliva shows potential for myasthenia gravis treatment

Looking for a better treatment for the autoimmune disease myasthenia gravis, researchers have found that a protein in tick saliva shows promise in limiting the severity of the disease in an animal model in a study published in the Annals of Neurology.

29 Mar 2009

Discovery of novel gene responsible for Lou Gehrig's disease

A team of Canadian and French researchers has identified a novel gene responsible for a significant fraction of ALS (sporadic amyotrophic lateral sclerosis) cases.

1 Apr 2008

Scientists study folding of the brain's cortex

Engineers at Washington University in St. Louis are finding common ground between the shaping of the brain and the heart during embryonic development.

2 Oct 2007

Stem cell therapy rescues motor neurons in ALS model

In a study that demonstrates the promise of cell-based therapies for diseases that have proved intractable to modern medicine, a team of scientists from the University of Wisconsin-Madison has shown it is possible to rescue the dying neurons characteristic of amyotrophic lateral sclerosis (ALS), a fatal neuromuscular disorder also known as Lou Gehrig's disease.

3 Aug 2007

Neuromuscular Disorder News and Research

TACT believes that Halo's HT-100 may effectively treat DMD

PTC, Roche announce licensing agreement for SMA programme

Researchers to begin drug development projects for rare and neglected diseases

Reduced levels of VAPB protein may play central role in causing ALS

RRD enters partnership with NIH Therapeutics for Rare and Neglected Diseases program

Research finds alteration in KBTBD13 protein causes nemaline myopathy

2010 Mid-Atlantic Bio a great kick-off for hosting 2011 BIO International Convention

ISIS-FXIRx and ISIS-SMNRx included in Isis Pharmaceuticals' development pipeline

UT physicians treat Iraqi girl with myasthenia gravis

Three genes linked to Lou Gehrig's disease

Safeway raises funds to support neuromuscular disease medical research and therapies

Muscular Dystrophy Canada to host the 44th Jerry Lewis Labour Day Telethon

Protein from tick saliva shows potential for myasthenia gravis treatment

Discovery of novel gene responsible for Lou Gehrig's disease

Scientists study folding of the brain's cortex

Stem cell therapy rescues motor neurons in ALS model

Stem cell therapy rescues motor neurons in amyotrophic lateral sclerosis model

Novel mutation associated with muscle identified

Groundbreaking research on type 1 congenital myotonic dystrophy

Researchers have discovered a genetic mutation associated with an inherited form of motor neuron disease

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