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).

FDA grants Orphan Drug designation to Milo Biotechnology's AAV1-FS344 inhibitor

Milo Biotechnology today announced its AAV1-FS344 has been granted Orphan Drug designation from the FDA's Office of Orphan Products Development for treatment of Becker and Duchenne muscular dystrophy. AAV1-FS344 is a gene therapy-delivered myostatin inhibitor that increases muscle strength.

12 Dec 2012

Longer looks: People with mental disabilities have a vote; Genome sequencing raises hope - also anxiety

I may get Alzheimer's disease, I was told after getting my DNA analyzed. I could suffer the consequences of an abnormal blood clot in my heart or my brain. Or perhaps I'll suffer from a disease called primary myelofibrosis that's caused by an overactive bone marrow. All of these were possible outcomes, based on my genetic profile. In each case, the test only gives an increased risk for disease, not a definitive diagnosis.

9 Nov 2012

‘Gene for speed’ researcher awarded Ramaciotti Medal

Professor Kathryn North AM, the researcher responsible for discovering the ‘gene for speed’, has been awarded the prestigious Ramaciotti Medal for Excellence in Biomedical Research and a $50,000 grant.

16 Oct 2012

Intensive treatment with high doses of statin drugs shows promise for dyslipidemia

A comprehensive new review on how to treat high cholesterol and other blood lipid problems suggests that intensive treatment with high doses of statin drugs is usually the best approach.

16 Oct 2012

New mouse model could foster ALS, Paget's and dementia breakthroughs

Efforts to treat disorders like Lou Gehrig's disease, Paget's disease, inclusion body myopathy and dementia will receive a considerable boost from a new research model created by UC Irvine scientists.

3 Oct 2012

New insights into filaminopathies

New insights into certain muscle diseases, the filaminopathies, are reported by an international research team led by Dr. Rudolf Andre Kley of the RUB's University Hospital Bergmannsheil in the journal Brain.

26 Sep 2012

New clinical trial to evaluate DEX-M74 against hereditary inclusion body myopathy

Researchers have launched a clinical trial to evaluate the drug candidate DEX-M74 as a treatment for a rare degenerative muscle disease, hereditary inclusion body myopathy (HIBM). National Institutes of Health scientists from the National Center for Advancing Translational Sciences (NCATS) and the National Human Genome Research Institute (NHGRI) will conduct the clinical trial at the NIH Clinical Center.

25 Sep 2012

NCI awards Soligenix $300,000 SBIR grant for orBec Phase 2 chronic GI GVHD trial

Soligenix, Inc., a development stage biopharmaceutical company, announced today that the National Cancer Institute has awarded Soligenix a Small Business Innovation Research grant to support the conduct of a Phase 2 clinical trial designed to evaluate orBec (oral beclomethasone 17,21-dipropionate or BDP) as a treatment for the gastrointestinal manifestation of chronic Graft-versus-Host disease.

5 Sep 2012

Statin's potency may cause muscle problems in patients with cardiovascular disease

A study from the University of California, San Diego School of Medicine, published August 22 online by PLoS ONE, reports that muscle problems reported by patients taking statins were related to the strength or potency of the given cholesterol-lowering drugs.

23 Aug 2012

Researchers discover new cause of congenital myopathy

University of Michigan researchers have discovered a new cause of congenital myopathy: a mutation in a previously uncharacterized gene, according to research published this month in the American Journal of Human Genetics.

26 Jul 2012

Lilly, Kowa announce results from PREVAIL U.S. study

Kowa Pharmaceuticals America, Inc. and Eli Lilly and Company today announced results of the PREVAIL U.S. study (Pitavastatin compaREd with praVAstatin In Lowering LDL-C in the U.S.) which evaluated the efficacy of LIVALO (pitavastatin) 4 mg compared with pravastatin 40 mg in reducing low-density lipoprotein cholesterol (LDL-C), the primary endpoint, as well as effects on other lipid parameters and lipoprotein particles in adult patients with primary hyperlipidemia or mixed dyslipidemia.

2 Jun 2012

BHD launches Statin Induced Myopathy Genotype test

Boston Heart Diagnostics (BHD) announced today the commercial launch of the Statin Induced Myopathy (SLCO1B1) Genotype test licensed from the University of Oxford's technology commercialization company Isis Innovation.

31 May 2012

Study identifies how skeletal muscle stem cells respond to muscle injury

A study conducted by Children's Hospital & Research Center Oakland scientists identifies how skeletal muscle stem cells respond to muscle injury and may be stimulated to improve muscle repair in Duchenne Muscular Dystrophy, a severe inherited disease of muscle that causes weakness, disability and, ultimately, heart and respiratory failure.

15 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

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

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

Patients worried about statin use after label warnings from FDA

The U.S. Food and Drug Administration's announcement Tuesday stated that it would require additional safety warnings on the labels of the cholesterol-lowering drugs. Doctors have said that while the benefits of statins outweigh their potential harm, some patients are alarmed by the new announcement.

1 Mar 2012

FDA grants orphan drug designation to Ultragenyx UX003 for treatment of MPS 7

Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced that the FDA Office of Orphan Products Development has granted orphan drug designation for UX003 for the treatment of MPS 7.

28 Feb 2012

Websites advertising statins for sale contain poor levels of safety information

A new study published in the journal Pharmacoepidemiology & Drug Safety reveals that internet sites selling prescription statins directly to consumers are widespread, and that most websites advertising statins for sale to the general public contain very poor levels of information relevant to safe use of the medicine and side effects.

3 Feb 2012

ACTF, Merck announce new HIV initiatives

Today, Merck, known as MSD outside the United States and Canada, and the ADAP Crisis Task Force (ACTF) announced a number of new initiatives to help struggling state AIDS Drug Assistance Programs (ADAPs) continue to provide access to medicines to people living with HIV.

22 Dec 2011