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Investigators in The Research Institute at Nationwide Children’s Hospital have identified a link between specific modifications of the dystrophin gene and the age of cardiac disease onset in patients with Becker muscular dystrophy (BMD). This information could help clinicians provide early cardiac intervention for BMD patients based on genetic testing results performed on a blood sample…

TCA Cellular Therapy, LLC (TCA-CT) announced that the U.S. Food and Drug Administration (FDA) has approved its adult stem cell protocol to conduct Phase I clinical trials to treat Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig’s disease)…

A team of University of British Columbia researchers has identified fat-producing cells that possess “dual-personalities” and may further the development of treatments for muscle diseases such as muscular dystrophy and fibrosis. The team found a new type of fibro/adipogenic progenitors, or FAPs, that generate fatty fibrous tissues when transplanted into damaged muscles in mice…

PTC Therapeutics, Inc. (PTC) announced the initiation of an additional clinical trial of ataluren (PTC124®) in boys and young men with nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) who have permanently lost the ability to walk independently. This trial is evaluating the best methods for measuring functional abilities in patients who have lost independent mobility…

Neuralstem, Inc. (NYSE Amex: CUR) announced that the first ALS patient was treated with its spinal cord stem cells yesterday at the Emory ALS Center at Emory University, in Atlanta, GA. A total of up to 18 patients is planned to be treated in this first U.S. clinical trial to evaluate human neural stem cells for the treatment of ALS (Amyotrophic Lateral Sclerosis, or Lou Gehrig’s disease)…

A study published in the online edition the American Journal of Human Genetics, allowed the first identification of a new form of adult onset muscular dystrophy. The research team led by Dr…

Research on the genetic defect that causes myotonic muscular dystrophy has revealed that the mutation disrupts an array of metabolic pathways in muscle cells through its effects on two key proteins…

As scientists work to find new treatments for Pompe disease – the devastating genetic “villain” that drives the efforts of the main characters in the new film “Extraordinary Measures” – University of Florida researchers are hopeful that gene therapy will help patients in the late stages of the disease breathe on their own…

Cytokinetics, Incorporated (NASDAQ: CYTK) announced results from its Phase I, randomized, double-blind, placebo-controlled, multiple-dose clinical trial of oral CK-2017357. The primary objective of this clinical trial was to determine the safety and tolerability of CK-2017357 after multiple oral doses to steady state in healthy male volunteers…

The ALS Therapy Development Institute and Oxford BioMedica announced today the extension of their collaboration following successful completion of the first phase…

California Stem Cell, Inc. (CSC) and ALS Therapy Development Institute (ALS TDI) are pleased to announce an extension and expansion of their collaboration aimed at advancing a potential stem cell therapy for ALS (amyotrophic lateral sclerosis)…

AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based drugs, announced that it received an orphan drug designation from the Committee for Orphan Medical Products of the European Medicines Agency (EMEA) for AVI-5038, a drug candidate being developed by AVI for the treatment of Duchenne Muscular Dystrophy (DMD). DMD is a genetic muscle-wasting disease caused by failure to produce dystrophin…

Regenerx Biopharmaceuticals, Inc. (NYSE Amex:RGN) announced today that a research team in Washington, D.C. has found that dystrophin-deficient Mdx mice, treated twice a week for six months with Tβ4, showed a significant increase in skeletal muscle regenerating fibers compared to untreated mice. No effects related to muscle function or fibrosis and no adverse reactions were observed in the mice…

Sandra Gidley MP is backing the fight for better muscle disease healthcare in Hampshire by calling on NHS bosses to fund specialist staff. Last month the South Central Muscle Group, a patients’ forum for families in the region living with muscular dystrophy, met in Southampton where MP for Romsey Sandra Gidley spoke to them about making their voices heard by politicians…

Clive Svendsen, Ph.D., director of the Cedars-Sinai Regenerative Medicine Institute, is receiving the American Academy of Neurology Sheila Essey Award for his research on ALS (amyothrophic lateral sclerosis). ALS, often referred to as Lou Gehrig’s disease, is a progressive neurodegenerative disease in which nerve cells called motor neurons degenerate in the brain and spinal cord…

A group of campaigning muscle disease patients from Yorkshire and Humber are proud to announce the Rt. Hon. William Hague MP as their new patron. The Yorkshire and Humber Muscle Group asked Mr Hague, as MP for Richmond and a Rotherham-born local lad, to be patron of their organisation and are pleased that he has accepted this post…

What Families affected by muscle disease in Nottingham and the East Midlands are meeting to hear how care and services in their region will be improved. Speaking at the meeting will be Kate Caston and Christine Richardson from the East Midlands NHS Specialised Commissioning Group…

Isis Pharmaceuticals, Inc. (Nasdaq: ISIS) announced that it has initiated a Phase 1 study of ISIS-SOD1Rx in patients with an inherited, aggressive form of Lou Gehrig’s disease also known as familial amyotrophic lateral sclerosis (ALS). Approximately 20 percent of all familial ALS cases are caused by a mutant form of superoxide dismutase, or SOD1…

Spinal Muscular Atrophy (SMA) is one of many serious disorders for which prenatal testing is available. SMA affects approximately 1 in 10,000 live births and is the leading genetic cause of infant mortality and the second most common autosomal recessive disorder, after cystic fibrosis…

What Families living with muscle disease in the East of England are joining forces with clinicians and MPs at an inaugural conference for the region, organised by the Muscular Dystrophy Campaign. The conference will be an opportunity for families to learn more about fighting for better muscle disease services in their region and to find out how local campaigning can make a real difference…

Prize4Life, an organization dedicated to finding a cure for amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease), is partnering with The Jackson Laboratory to help more researchers identify treatment candidates that increase the lifespan of mice that model ALS…

Neuralstem, Inc. (NYSE Amex: CUR) announced that its Phase I trial to treat Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig’s disease) with its spinal cord stem cells has been approved by the Institutional Review Board (IRB) at Emory University in Atlanta, GA. The trial, which was approved by the FDA in September, will take place at the Emory ALS Center, under the direction of Dr…

With the discovery of a new type of chemical modification on an important muscle protein, a University of Iowa study improves understanding of certain muscular dystrophies and could potentially lead to new treatments for the conditions. The findings, which appear in the Jan. 1, 2010, issue of the journal Science, may also have implications for detecting metastasizing cancer cells…

University at Buffalo biophysicists have found a protein in tarantula venom that shows promise as a potential therapy for muscular dystrophy (MD). They have formed a start-up biotech company in Buffalo — Rose Pharmaceuticals — to advance the drug to clinical trials…

A drug already used to treat symptoms of epilepsy has potential to slow the muscle weakening that comes with amyotrophic lateral sclerosis (ALS), scientists report after completing a Phase II clinical trial-an early, small-scale test to show if the drug works and continues to be safe…

Buoyed by the extraordinary progress being made by the ALS Therapy Development Institute, the Muscular Dystrophy Association today announced a new milestone-driven grant of $2.5 million, adding to the $18 million MDA already has invested with ALS TDI — the world’s only non-profit research center focused exclusively on developing treatments for amyotrophic lateral sclerosis (ALS)…

A study appearing in Science Translational Medicine puts scientists one step closer to clinical trials to test a gene delivery strategy to improve muscle mass and function in patients with certain degenerative muscle disorders. Severe weakness of the quadriceps is a defining feature of several neuromuscular disorders…

McClatchy/The Miami Herald reports on Ian Pearl, a South Florida man born with muscular dystrophy who is drawing attention to discriminatory health insurance practices against the disabled…

Tesco customers are being asked to get dunking to raise at least £1,500 for children with muscle-wasting diseases. The retailer has created this limited edition biscuit exclusively to raise funds for the Muscular Dystrophy Campaign, Tesco Charity of the Year 2009…

A baby with a rare neuromuscular condition who has been on a ventilator in hospital since birth, known for legal reasons as Baby RB, has been at the centre of a legal debate between his parents – a debate which has now ended with the baby’s father withdrawing his objections to the ventilator being switched off…

When muscle cells need repair, they use odor-detecting tools found in the nose to start the process, researchers have discovered. The results are published online and scheduled for publication in the November issue of the journal Developmental Cell Found on the surfaces of neurons inside the nose, odorant receptors are molecules that bind and respond to substances wafting through the air…

While previous studies have indicated a “probable” connection between smoking and ALS, a new study published in the Nov. 17, 2009 issue of Neurology®, the medical journal of the American Academy of Neurology, states that smoking may now be considered an “established” risk factor for Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease…

New discoveries have been made about how an elevated level of lead, which is a neurotoxic heavy metal, can slow the progression of amyotrophic lateral sclerosis, or Lou Gehrig’s disease – findings that could point the way to a new type of therapy. The results surprised researchers, since lead is also a known risk factor for ALS…

It turns out that wearing a cap is good for you, at least if you are a mammal cell. Researchers from the Johns Hopkins Engineering in Oncology Center have shown that in healthy cells, a bundled “cap” of thread-like fibers holds the cell’s nucleus, its genetic storehouse, in its proper place…

CytRx Corporation (NASDAQ:CYTR), a biopharmaceutical company, is permitted to re-enter the clinic with its orally administered molecular chaperone drug candidate arimoclomol as a therapeutic treatment for amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease), following the U.S. Food and Drug Administration’s (FDA) acceptance of a revised clinical trial protocol…

Humans and mice have previously unknown and potentially critical differences in one of the genes responsible for Duchenne muscular dystrophy (DMD)…

Data published online in the medical journal Muscle and Nerve support the use of the six-minute walk test (6MWT) as an outcome measure in PTC Therapeutics, Inc.’s ongoing registration-directed clinical trial of ataluren in patients with nonsense mutation Duchenne/Becker muscular dystrophy (nmDMD/BMD)…

Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced the presentation of preliminary data from the Phase 2 clinical trial of its ZFP Therapeutic(TM) program to develop SB-509 as a treatment for amyotrophic lateral sclerosis (ALS) at a Work in Progress session at the 20th International Symposium on ALS/MND held in Berlin, Germany…

Knopp Neurosciences Inc. (”Knopp”) announced the presentation of encouraging clinical results in a Phase 2 safety and tolerability study of KNS-760704 in amyotrophic lateral sclerosis (”ALS”). The results were presented at the 20th International Symposium on ALS/MND in Berlin, Germany, by Merit Cudkowicz, M.D…

Researchers at UT Southwestern Medical Center have found that a molecule produced naturally by muscles in response to nerve damage can reduce symptoms and prolong life in a mouse model of amyotrophic lateral sclerosis (ALS). “We believe we can apply this research toward drug development,” said Dr…

Data published online in the medical journal Muscle and Nerve support the use of the six-minute walk test (6MWT) as an outcome measure in PTC Therapeutics, Inc.’s ongoing registration-directed clinical trial of ataluren in patients with nonsense mutation Duchenne/Becker muscular dystrophy (nmDMD/BMD)…

Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced the presentation of preliminary data from the Phase 2 clinical trial of its ZFP Therapeutic(TM) program to develop SB-509 as a treatment for amyotrophic lateral sclerosis (ALS) at a Work in Progress session at the 20th International Symposium on ALS/MND held in Berlin, Germany…

New discoveries have been made about how an elevated level of lead, which is a neurotoxic heavy metal, can slow the progression of amyotrophic lateral sclerosis, or Lou Gehrig’s disease – findings that could point the way to a new type of therapy. The results surprised researchers, since lead is also a known risk factor for ALS…

It turns out that wearing a cap is good for you, at least if you are a mammal cell. Researchers from the Johns Hopkins Engineering in Oncology Center have shown that in healthy cells, a bundled “cap” of thread-like fibers holds the cell’s nucleus, its genetic storehouse, in its proper place…

CytRx Corporation (NASDAQ:CYTR), a biopharmaceutical company, is permitted to re-enter the clinic with its orally administered molecular chaperone drug candidate arimoclomol as a therapeutic treatment for amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease), following the U.S. Food and Drug Administration’s (FDA) acceptance of a revised clinical trial protocol…

Humans and mice have previously unknown and potentially critical differences in one of the genes responsible for Duchenne muscular dystrophy (DMD)…

Tesco customers are being asked to get dunking to raise at least £1,500 for children with muscle-wasting diseases. The retailer has created this limited edition biscuit exclusively to raise funds for the Muscular Dystrophy Campaign, Tesco Charity of the Year 2009.

A baby with a rare neuromuscular condition who has been on a ventilator in hospital since birth, known for legal reasons as Baby RB, has been at the centre of a legal debate between his parents – a debate which has now ended with the baby’s father withdrawing his objections to the ventilator being switched off. He was born with congenital myasthenic syndrome, a muscle weakness that limits the movement of his limbs and his ability to breathe on his own.

When muscle cells need repair, they use odor-detecting tools found in the nose to start the process, researchers have discovered. The results are published online and scheduled for publication in the November issue of the journal Developmental Cell Found on the surfaces of neurons inside the nose, odorant receptors are molecules that bind and respond to substances wafting through the air.

While previous studies have indicated a “probable” connection between smoking and ALS, a new study published in the Nov. 17, 2009 issue of Neurology®, the medical journal of the American Academy of Neurology, states that smoking may now be considered an “established” risk factor for Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease. The findings come from Baystate Medical Center neurologist Dr.