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Isis Pharmaceuticals begins ISIS-DMPK Rx clinical study in DM1 patients

Isis Pharmaceuticals begins ISIS-DMPK Rx clinical study in DM1 patients

Isis Pharmaceuticals, Inc. announced today that it has initiated a study for ISIS-DMPK Rx in patients with Myotonic Dystrophy Type 1 (DM1). DM1 is a rare genetic neuromuscular disease caused by the production of toxic dystrophia myotonica-protein kinase (DMPK) RNA in cells. ISIS-DMPKRx is specifically designed to reduce toxic DMPK RNA. [More]
Isis Pharmaceuticals CEO honored with SCRIP Lifetime Achievement Award

Isis Pharmaceuticals CEO honored with SCRIP Lifetime Achievement Award

Stanley T. Crooke, Ph.D., M.D., founder, CEO and chairman of the board of directors of Isis Pharmaceuticals, Inc., has been awarded the 2014 SCRIP Lifetime Achievement Award, one of the most prestigious of its kind in the pharmaceutical and biotechnology industries worldwide. [More]
New study examines efficacy of brace application for treatment of spinal compression fractures

New study examines efficacy of brace application for treatment of spinal compression fractures

Compression fractures in the spine due to osteoporosis, a common condition causing progressive bone loss and increased fracture risk, are especially common in older women. A new study appearing in the December 3rd issue of the Journal of Bone & Joint Surgery found that patients who wore a brace as treatment for a spinal compression fracture had comparable outcomes in terms of pain, function and healing when compared to patients who did not wear a brace. [More]
Isis Pharmaceuticals announces initiation of ISIS-SMN Rx Phase 3 study in children with SMA

Isis Pharmaceuticals announces initiation of ISIS-SMN Rx Phase 3 study in children with SMA

Isis Pharmaceuticals, Inc. announced today the initiation of a pivotal Phase 3 study evaluating ISIS-SMNRx in approximately 120 non-ambulatory children with spinal muscular atrophy (SMA). [More]
Isis receives $10 million from Biogen Idec to initiate IND-supporting studies of ISIS-BIIB3 Rx

Isis receives $10 million from Biogen Idec to initiate IND-supporting studies of ISIS-BIIB3 Rx

Isis Pharmaceuticals, Inc. announced today that it has earned a $10 million milestone payment from Biogen Idec related to the initiation of investigational new drug (IND)-supporting studies of ISIS-BIIB3Rx. ISIS-BIIB3Rx is a recently identified development candidate designed to inhibit an undisclosed target to treat a neurodegenerative disease. [More]
Murdoch University researchers receive NHMRC grant to develop genetic drugs for rare diseases

Murdoch University researchers receive NHMRC grant to develop genetic drugs for rare diseases

Murdoch University researchers from the Centre for Comparative Genomics have received almost $800,000 in funding from the National Health and Medical Research Council to develop genetic drugs to treat rare diseases. [More]
Growth factor boosts natural defence against auto-immune diseases

Growth factor boosts natural defence against auto-immune diseases

Our immune system defends us from harmful bacteria and viruses, but, if left unchecked, the cells that destroy those invaders can turn on the body itself, causing auto-immune diseases like type-1 diabetes or multiple sclerosis. A molecule called insulin-like growth factor-1 (IGF-1) boosts the body's natural defence against this 'friendly fire', scientists at the European Molecular Biology Laboratory in Monterotondo, Italy, have found. [More]
Isis Pharmaceuticals provides update on Phase 2 clinical studies of ISIS-SMN Rx in children with SMA

Isis Pharmaceuticals provides update on Phase 2 clinical studies of ISIS-SMN Rx in children with SMA

Isis Pharmaceuticals, Inc. today provided an update on its ongoing open-label Phase 2 clinical studies of ISIS-SMN Rx in infants and children with spinal muscular atrophy at the 19th International World Muscle Society (WMS) Congress in Berlin, Germany. [More]
Cedars-Sinai to participate in a consortium studying motor neuron disorders

Cedars-Sinai to participate in a consortium studying motor neuron disorders

Investigators at the Cedars-Sinai Board of Governors Regenerative Medicine Institute have received a grant from the National Institutes of Health to participate in a consortium taking the study of motor neuron disorders - such as Lou Gehrig's disease and spinal muscular atrophy - to a new, comprehensive perspective. [More]
MU researchers make new breakthrough in spinal muscular atrophy drug

MU researchers make new breakthrough in spinal muscular atrophy drug

According to recent studies, approximately one out of every 40 individuals in the United States is a carrier of the gene responsible for spinal muscular atrophy (SMA), a neurodegenerative disease that causes muscles to weaken over time. [More]
Six research institutions awarded NIH grants to create database of human cellular responses

Six research institutions awarded NIH grants to create database of human cellular responses

Building on a successful three-year pilot project, the National Institutes of Health has awarded more than $64 million to six research institutions to create a database of human cellular responses, the Library of Integrated Network-based Cellular Signatures. [More]
Scientists identify mechanism by which inherited neurological disease causes muscle weakness in men

Scientists identify mechanism by which inherited neurological disease causes muscle weakness in men

Researchers at University of California, San Diego School of Medicine have identified the mechanism by which a rare, inherited neurodegenerative disease causes often crippling muscle weakness in men, in addition to reduced fertility. [More]
Splice-switching oligonucleotide drugs alter editing of gene transcript

Splice-switching oligonucleotide drugs alter editing of gene transcript

In splice-switching, an innovative therapeutic approach, targeted oligonucleotide drugs alter the editing of a gene transcript to produce the desired form of a protein. [More]
Isis Pharmaceuticals begins ISIS-DMPK Rx Phase 1 study for treatment of Myotonic Dystrophy Type 1

Isis Pharmaceuticals begins ISIS-DMPK Rx Phase 1 study for treatment of Myotonic Dystrophy Type 1

Isis Pharmaceuticals, Inc. announced today that it has initiated a Phase 1 study for ISIS-DMPKRx. Isis earned a $14 million milestone payment from Biogen Idec associated with this achievement. [More]
Study: Sufficient copies of SMN1 gene extend survival in animals with spinal muscular atrophy

Study: Sufficient copies of SMN1 gene extend survival in animals with spinal muscular atrophy

To make up for insufficient amounts of SMN protein, the cause of the inherited neuromuscular disease spinal muscular atrophy (SMA), researchers have successfully delivered a replacement SMN1 gene directly to the spinal cords of animal models of SMA. [More]
Isis Pharmaceuticals reports pro forma net operating loss of $22.6 million for Q1 2014

Isis Pharmaceuticals reports pro forma net operating loss of $22.6 million for Q1 2014

Isis Pharmaceuticals, Inc. today reported a pro forma net operating loss (NOL) of $22.6 million for the three months ended March 31, 2014 compared to pro forma operating income of $4.5 million for the same period in 2013. [More]
Astrocytes malfunction may contribute to neurodegenerative disorders

Astrocytes malfunction may contribute to neurodegenerative disorders

Scientists studying brain diseases may need to look beyond nerve cells and start paying attention to the star-shaped cells known as "astrocytes," because they play specialized roles in the development and maintenance of nerve circuits and may contribute to a wide range of disorders, according to a new study by UC San Francisco researchers. [More]
Positive results from pivotal clinical trial of Trophos’ olesoxime in SMA patients to be presented at AAN 2014

Positive results from pivotal clinical trial of Trophos’ olesoxime in SMA patients to be presented at AAN 2014

Trophos and AFM-Telethon today announce that data from the pivotal clinical trial of Trophos’ lead product candidate olesoxime in spinal muscular atrophy (SMA) will be presented during the 66th American Academy of Neurology annual meeting to be held in Philadelphia, PA, USA, from April 26 to May 3 2014. [More]
New mouse study indicates that mutant protein in muscle cells is responsible for SBMA

New mouse study indicates that mutant protein in muscle cells is responsible for SBMA

Sometimes known as Kennedy's disease, spinal and bulbar muscular atrophy (SBMA) is a rare inherited neuromuscular disorder characterized by slowly progressive muscle weakness and atrophy. [More]
New way to make large concentrations of skeletal muscle cells from human stem cells

New way to make large concentrations of skeletal muscle cells from human stem cells

As stem cells continue their gradual transition from the lab to the clinic, a research group at the University of Wisconsin-Madison has discovered a new way to make large concentrations of skeletal muscle cells and muscle progenitors from human stem cells. [More]