Neuromuscular Disease News and Research

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Laminin-111 protein therapy could treat Duchenne muscular dystrophy

A novel treatment in development at the University of Nevada School of Medicine for the most common form of muscular dystrophy is advancing towards human trials with a $308,000 boost from the Muscular Dystrophy Association.

23 Aug 2012

UI Hospital implants NeuRx Diaphragm Pacing System in a patient with ALS

The University of Illinois Hospital & Health Sciences System is one of only a dozen hospitals nationwide to implant the NeuRx Diaphragm Pacing System in a patient with amyotrophic lateral sclerosis (ALS).

3 Aug 2012

Advanced understanding of neurodegenerative diseases achieved by Trophos

Trophos SA, a clinical stage pharmaceutical company developing innovative therapeutics from discovery to clinical validation for indications with under-served needs in neurology and cardiology, announces today the conclusion of the pan-European MitoTarget Project, the EU funded orphan disease project, with submission of the final report to the EC.

31 Jul 2012

FDA approves Baxter's GAMMAGARD LIQUID for multifocal motor neuropathy

Baxter International Inc. today announced that the U.S. Food and Drug Administration (FDA) has approved GAMMAGARD LIQUID 10% [Immune Globulin Infusion (Human)] as a treatment for multifocal motor neuropathy (MMN). This is the first immunoglobulin treatment approved for MMN patients in the United States, and it was approved for use with MMN patients in Europe in 2011.

From Baxter International Inc. 25 Jun 2012

Diabetic Neuropathy: An interview with Dr Brian Callaghan

Diabetes is the number 1 cause of neuropathy, which is a very common condition in itself. In fact, up to half of all neuropathy is caused by diabetes, although other people can get the condition as well.

25 Jun 2012

New understanding of how motor neurons die in spinal muscular atrophy patients

Cedars-Sinai's Regenerative Medicine Institute has pioneered research on how motor-neuron cell-death occurs in patients with spinal muscular atrophy, offering an important clue in identifying potential medicines to treat this leading genetic cause of death in infants and toddlers.

21 Jun 2012

DYNC1H1 genetic mutation contributes to SMA-LED

Scientists have confirmed that mutations of a gene are responsible for some cases of a rare, inherited disease that causes progressive muscle degeneration and weakness: spinal muscular atrophy with lower extremity predominance, also known as SMA-LED.

11 May 2012

Research grants on facioscapulohumeral dystrophy prove useful as genetic basis is found

Businessman Bill Moss’s determination to fight his untreatable muscle-wasting disease may have paid off. Four years ago, Mr Moss, a former Macquarie Bank Executive Director, established a charitable research foundation, FSHD Global, which partly funded the Italian researchers who made the breakthrough.

8 May 2012

Ultragenyx reports results from UX001 Phase 1 study on HIBM

Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced results from a first-in-human, multi-center, Phase 1 study of UX001 in patients with hereditary inclusion body myopathy (HIBM) showing that UX001 was well-tolerated with an expected extended release profile on absorption after oral administration

2 May 2012

UIC researcher to explore causes of myasthenia gravis

A University of Illinois at Chicago researcher is the recipient of a $240,000 research grant to further explore the causes of myasthenia gravis, a neurologic disease affecting muscles.

27 Apr 2012

Positive results from Repligen's RG3039 Phase 1 study for spinal muscular atrophy

Repligen Corporation today announced positive results from a Phase 1 study to evaluate the pharmacokinetic (PK) and safety profile of RG3039, a novel small molecule drug candidate for the potential treatment of spinal muscular atrophy (SMA).

25 Apr 2012

Genetically modified mouse mimics key features of Charcot-Marie-Tooth disease

A team of scientists from the University of Missouri created a genetically modified mouse that mimics key features of Charcot-Marie-Tooth disease, an inherited neuromuscular disease affecting approximately 150,000 people in the United States.

18 Apr 2012

EMA grants orphan designations to Ultragenyx UX001, UX003 for treatment of HIBM and MPS 7

Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced that the European Medicines Agency (EMA) has granted orphan drug designations for sialic acid for the treatment of hereditary inclusion body myopathy (HIBM) and recombinant human beta-glucuronidase for the treatment of mucopolysaccharidosis type 7.

29 Mar 2012

Invasive strategy benefits patients with myotonic dystrophy type 1 neuromuscular disorder

Patients with a cardiac irregularity and myotonic dystrophy type 1 (a severe neuromuscular disorder with a high risk of sudden death) who received an invasive treatment strategy that included testing of their heart's electrical conduction system and if needed, implantation of a device such as a pacemaker, had an associated higher rate of 9-year survival compared to patients treated noninvasively, according to a study in the March 28 issue of JAMA.

28 Mar 2012

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

Ultragenyx completes UX001 Phase 1 study on hereditary inclusion body myopathy

Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced that the Phase 1 study of UX001 for hereditary inclusion body myopathy (HIBM) has been completed.

9 Mar 2012

Prosensa secures €23M in new equity financing

Prosensa, the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet needs, announced today that it has raised €23 million in new equity financing.

25 Jan 2012

Four Loyola anesthesiologists have written chapters for Manual of Clinical Anesthesiology

Four Loyola anesthesiologists have written chapters for the Manual of Clinical Anesthesiology, a popular reference and teaching tool used in hospitals across the nation.

13 Jan 2012

Immunosuppressant combination improves survival of human spinal stem cells in ALS rat model

A team of researchers grafting human spinal stem cells into rats modeled with amyotrophic lateral sclerosis (ALS), also known as "Lou Gehrig's Disease," a degenerative, lethal, neuromuscular disease, have tested four different immunosuppressive protocols aimed at determining which regimen improved long-term therapeutic effects.

20 Dec 2011

People with myotonic muscular dystrophy more likely to develop cancer

People who have the most common type of adult muscular dystrophy also have a higher risk of getting cancer, according to a paper published today in the Journal of the American Medical Association.

14 Dec 2011