Myopathy News and Research

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The myopathies are neuromuscular disorders in which the primary symptom is muscle weakness due to dysfunction of muscle fiber. Other symptoms of myopathy can include include muscle cramps, stiffness, and spasm. Myopathies can be inherited (such as the muscular dystrophies) or acquired (such as common muscle cramps).

Researchers unlock structural details of key protein to treat neuromuscular disease

Researchers at Rice University and Baylor College of Medicine have unlocked the structural details of a protein seen as key to treating a neuromuscular disease.

30 Sep 2015

Research highlights potential new treatment strategy for degenerative diseases

St. Jude Children's Research Hospital scientists have discovered evidence of a mechanism at the heart of amyotrophic lateral sclerosis (ALS) and related degenerative diseases. The research appears in today's edition of the journal Cell and highlights a possible new treatment strategy for the devastating disorders.

25 Sep 2015

First patient dosed in long term-safety extension study of Resolaris for adult patients with FSHD

aTyr Pharma, Inc., a biotherapeutics company engaged in the discovery and development of Physiocrine-based therapeutics to address severe rare diseases, today announced that the first patient has been dosed in a newly initiated long term-safety extension study of Resolaris for adult patients with facioscapulohumeral muscular dystrophy (FSHD), a rare and severe genetic myopathy for which there are currently no approved treatments.

10 Sep 2015

Scientists reveal role of dynamin and show how specific mutations impair function of dynamin

Cells continually form membrane vesicles that are released into the cell. If this vital process is disturbed, nerve cells, for example, cannot communicate with each other. The protein molecule dynamin is essential for the regulated formation and release of many vesicles.

28 Aug 2015

Clementia expands enrollment to include children with FOP in ongoing Phase 2 clinical trial

Clementia Pharmaceuticals, Inc. today announced the initiation of enrollment of children as young as 6 years old in the company's ongoing Phase 2 study of palovarotene for the treatment of fibrodysplasia ossificans progressiva (FOP).

26 Aug 2015

Necrotising autoimmune myopathy requires early, aggressive treatment

Researchers call for the prompt recognition of necrotising autoimmune myopathy and aggressive early treatment with a combination of intravenous immune globulin, corticosteroids and a steroid-sparing agent for 3 months.

27 Jul 2015

Substantial pharmacogenetic information available on cardiovascular drugs

There is a wealth of published information describing interactions between drugs used to treat cardiovascular disease and the genetic variations that can affect how patients respond to them. But few heart specialists make routine use of this potentially life-saving data.

2 Jun 2015

Clementia Pharmaceuticals commences multi-center study of patients with fibrodysplasia ossificans progressiva

Clementia Pharmaceuticals, Inc. today announced that it has commenced enrollment in the second part (Part B) of its natural history study in patients with fibrodysplasia ossificans progressiva (FOP), a rare, severely disabling congenital myopathy characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) that result in the formation of new, abnormal (heterotopic) bone in muscles, tendons and ligaments.

28 May 2015

aTyr's Resolaris granted FDA Orphan Drug Designation for treatment of FSHD

aTyr Pharma, a biotherapeutics company engaged in the discovery and development of Physiocrine-based therapeutics to address rare diseases, announced today that Resolaris has been granted Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of facioscapulohumeral muscular dystrophy (FSHD).

27 Apr 2015

Joseph Roche receives grant to develop improved exercise programs for people with dysferlinopathies

The Wayne State University Eugene Applebaum College of Pharmacy and Health Sciences today announced that Joseph Roche, assistant professor for the physical therapy program in the Department of Health Care Sciences, received a one-year, $100,000 grant from the Jain Foundation for "Developing a Multi-Pronged Strategy for the Clinical Management of Dysferlinopathies," a project that seeks to develop better exercise programs for people with dysferlinopathies, or dysferlin-linked muscular dystrophies.

21 Apr 2015

TGen scientists discover the likely cause of rare type of muscle weakness in six children

Scientists at the Translational Genomics Research Institute (TGen), using state-of-the-art genetic technology, have discovered the likely cause of a child's rare type of severe muscle weakness.

10 Apr 2015

EC grants orphan drug designation to aTyr Pharma's Resolaris for treatment of FSHD

aTyr Pharma, Inc., a biotherapeutics company engaged in the discovery and development of Physiocrine-based therapeutics to address rare diseases, announced today the European Commission (EC) has granted orphan drug designation to Resolaris for the treatment of facioscapulohumeral muscular dystrophy (FSHD).

19 Feb 2015

Clementia secures additional $10 million to support development of palovarotene for treatment of FOP

Clementia Pharmaceuticals, Inc. today announced that it has secured an additional $10 million from current investors to support development of the company's lead compound palovarotene for the treatment of fibrodysplasia ossificans progressive (FOP).

5 Jan 2015

AutoGenomics, Genomas sign agreement to develop DNA-guided diagnostic systems

AutoGenomics, Inc, a leader in automated microarray technology for molecular diagnostics, and Genomas, a biomedical company enabling DNA-guided medicine for personal healthcare, announced today that they have signed an agreement to jointly develop genetic tests and DNA-guided diagnostic systems for optimal selection of statins and for improved delivery of statin therapy for the treatment of cardiovascular disease, obesity and diabetes.

7 Jul 2014

Merck enrolls first patient in reformulated raltegravir Phase 3 trial for treatment of HIV-1 infection

Merck, known as MSD outside the United States and Canada, today announced that the first patient has been enrolled in the company's global Phase 3 clinical trial, ONCEMRK.

6 Jun 2014

Researchers show that vitamin B3 can slow down progression of mitochondrial disease

The researchers of the University of Helsinki, Finland, and école Polytechnique Fédérale de Lausanne, Switzerland, have shown that vitamin B3 form nicotinamide riboside can slow down the progression of mitochondrial disease, suggesting its potential as a novel therapy approach to adult-onset mitochondrial muscle diseases.

7 Apr 2014

Jain Foundation's dysferlinopathy clinical study exceeds original goal of 150 patients

The Jain Foundation is delighted to announce that 193 patients have been recruited into the Clinical Outcome Study for Dysferlinopathy (COS), exceeding the original goal of 150 patients.

2 Apr 2014

Investigators urge drug regulators to provide clear evidence on statins’ side effects

At a time when the wider prescription of statins is under renewed public scrutiny, a substantial analysis of placebo-controlled randomised trials of statins has found that only a small minority of side effects reported by those taking the cholesterol-lowering drugs are actually attributable to them.

13 Mar 2014

Patients with congenital disorders are urged to register with CMDIR to advance clinical trials

If you are affected by Malignant Hyperthermia, Congenital Muscular Dystrophies or Congenital Myopathy Subtypes, the Malignant Hyperthermia Association of the United States and the Cure Congenital Muscle Disease organization are asking for your help to enable research and clinical trials by registering with the Congenital Muscle Disease International Registry (CMDIR).