Neuromuscular Disease News and Research

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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

ACGME approves Cedars-Sinai's neurology residency training program

The Accreditation Council for Graduate Medical Education has approved a neurology residency training program at Cedars-Sinai Medical Center. Recruitment of the first group of three residents is under way through the 2012 "match," a process in which an independent organization pairs applicants with programs to meet the preferences of each.

30 Nov 2011

Health supplement hope for child muscle disease

A health supplement used by bodybuilders could be the key to treating a life-threatening muscular dystrophy affecting hundreds of Australian children, new research shows.

14 Nov 2011

Cancer cells maintain rapid proliferation due to PK-M2 enzyme

Cancer cells maintain their life-style of extremely rapid growth and proliferation thanks to an enzyme called PK-M2 (pyruvate kinase M2) that alters the cells' ability to metabolize glucose - a phenomenon known as the Warburg effect.

3 Nov 2011

People with Parkinson's can benefit from computer-based physical therapy games

Playing computer-based physical therapy games can help people with Parkinson's disease improve their gait and balance, according to a new pilot study led by the UCSF School of Nursing and Red Hill Studios, a California serious games developer.

20 Oct 2011

FARA announces 2011 Ataxian Athlete Initiative grant recipients

The Friedreich's Ataxia Research Alliance (FARA), in partnership with The FA Project, Catrike, Anna Maria Oyster Bar, and The Melting Pot are pleased to announce Bill Ramsey, Tom Trovinger, Mary Vida, and Patrick Cogan as the 2011 Ataxian Athlete Initiative (AAI) grant recipients.

10 Oct 2011

FDA approves NeuRx Diaphragm Pacing System to treat ALS

An electronic system that stimulates the nerve of the diaphragm muscles has received approval from the Food and Drug Administration (FDA) for use in patients with Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease.

30 Sep 2011

Researchers identify common genetic mutation for ALS, frontotemporal dementia

Frontotemporal dementia and amyotrophic lateral sclerosis, also known as Lou Gehrig's disease -- two fatal neurodegenerative disease with distinct but sometimes overlapping symptoms -- are triggered by a common mutation in many cases, according to researchers who say they have identified the mutated gene.

22 Sep 2011

Researchers’ discovery may revolutionize treatment of Lou Gehrig’s disease

A team of researchers from the University of British Columbia and the Vancouver Coastal Health Research Institute have found a key link between prions and the neurodegenerative disease ALS (Amyotrophic Lateral Sclerosis), also known as Lou Gehrig’s disease. The discovery is significant as it opens the door to novel approaches to the treatment of ALS.

20 Sep 2011

Encouraging results from Alexion's eculizumab Phase 2 study on generalized myasthenia gravis

Alexion Pharmaceuticals, Inc. today announced that the Company's study of eculizumab in a small group of 14 patients with severe and refractory generalized myasthenia gravis, an ultra-rare and debilitating form of generalized myasthenia gravis (gMG), showed a strong disease improvement signal.

15 Sep 2011

Walgreens, MDA team up to offer free seasonal flu shots

Nobody likes to get stuck with a needle. But influenza - the flu - is particularly hazardous for those with neuromuscular disease, which is why the Muscular Dystrophy Association (MDA) and Walgreens have teamed up once again to offer free seasonal flu shots now underway for people who have muscular dystrophy and related diseases.

14 Sep 2011