Neuromuscular Disease News and Research

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Trophos' olesoxime shows beneficial effect on maintaining motor function in SMA patients

Trophos today announces that top-line results from a pivotal clinical trial of its lead product candidate olesoxime in spinal muscular atrophy (SMA) show a beneficial effect on the maintenance of motor function in SMA patients. If approved, olesoxime could be the first treatment specifically developed for SMA patients.

10 Mar 2014

Scientists receive new awards from CIRM to advance revolutionary stem cell science in medicine

Scientists from UCLA's Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research have received new awards from the California Institute of Regenerative Medicine (CIRM), the state stem cell research agency, that will forward revolutionary stem cell science in medicine.

30 Jan 2014

ALS TDI forms research partnership with Neurimmune to advance potential treatments for ALS

The ALS Therapy Development Institute (ALS TDI), together with its wholly-owned subsidiary, Anelixis Therapeutics, announced today that it has formed a research partnership with Neurimmune to advance potential treatments for ALS, also known as Lou Gehrig's disease.

6 Dec 2013

Imaginative play with medically themed toys may improve recovery in chronically ill children

New research finds that chronically ill children celebrate a successful recovery. It's through their imaginative play with medically themed toys. Laura Nabors, an associate professor of human services in the University of Cincinnati College of Education, Criminal Justice, and Human Services (CECH), will present new research on Tuesday, Nov. 5, at the American Public Health Association's (APHA) 141st Annual Meeting and Expo in Boston.

6 Nov 2013

Trophos completes efficacy study of olesoxime in patients with Spinal Muscular Atrophy

Trophos SA, a clinical stage pharmaceutical company developing innovative therapeutics from discovery to clinical validation for indications with under-served needs in neurology and cardiology, has completed its efficacy study of olesoxime in the rare neurodegenerative condition Spinal Muscular Atrophy.

5 Nov 2013

Three Penn Medicine researchers awarded grants for neuromuscular disease research

The Muscular Dystrophy Association has awarded research grants totaling over $1,000,000 to three Penn Medicine researchers: James Shorter, PhD, associate professor of Biochemistry and Biophysics; Hansell Stedman, MD, associate professor of Surgery; and Lee Sweeney, PhD, director of the Center for Orphan Disease Research and Therapy.

9 Oct 2013

Quintiles and MDA partner to develop U.S. Neuromuscular Disease Registry

Quintiles and the Muscular Dystrophy Association (MDA) today announced a new partnership to develop and implement the U.S. Neuromuscular Disease Registry, a patient registry that will play an important role in determining effective treatments for people with muscular dystrophy and related muscle diseases.

9 Oct 2013

Isis Pharmaceuticals announces preliminary data from ISIS-SMNRx Phase 1 study in infants with SMA

Isis Pharmaceuticals, Inc. announced today that follow-up preliminary data from a single dose, open-label Phase 1 study of ISIS-SMNRx in children with spinal muscular atrophy, show that most SMA children receiving the two highest doses of the drug (6 mg and 9 mg) continued to show improvements in muscle function tests up to 14 months after a single injection of the drug.

19 Sep 2013

Isis Pharmaceuticals starts ISIS-SMNRx Phase 2 study in infants with SMA

Isis Pharmaceuticals, Inc. announced the initiation of a Phase 2 study of ISIS-SMNRx in infants with spinal muscular atrophy. SMA is a severe and rare genetic neuromuscular disease characterized by muscle atrophy and weakness and is the most common genetic cause of infant mortality.

24 Apr 2013

DART Therapeutics develops new class of therapy for Duchenne muscular dystrophy

DART Therapeutics Inc., an innovative, new-model biotechnology firm focused on developing therapies for Duchenne muscular dystrophy, announced today that it is developing a SARM drug candidate obtained from Belgium-based Galapagos NV.

21 Mar 2013

Genetic testing services for rare neurological disorders launched by Athena Diagnostics

Athena Diagnostics, a leader in neurological diagnostics, today announced the clinical availability of new genetic tests to aid the detection of several rare neurological disorders, including hereditary neuropathy, neuromuscular disease, epilepsy and certain movement disorders. The lab-developed tests are available through Athena Diagnostics, a business of Quest Diagnostics (NYSE:DGX), the world’s leading provider of diagnostic information services.

From Quest Diagnostics 19 Mar 2013

Dementia diagnostic panel for treatable forms of cognitive impairment launched by Quest Diagnostics

Quest Diagnostics (NYSE: DGX), the world’s leading provider of diagnostic information services, announced the availability of the first clinical test panel for aiding the diagnosis of suspected dementia due to treatable forms of cognitive impairment. The test panel is believed to be the first commercial service from a clinical laboratory to combine several guideline-recommended tests for identifying secondary, treatable causes of dementia as a single blood test and report.

From Quest Diagnostics 19 Mar 2013

Columbia University Medical Center, NewYork-Presbyterian Hospital present research works at AAN meeting

The following research from Columbia University Medical Center and NewYork-Presbyterian Hospital is being presented at the 65th annual meeting of the American Academy of Neurology, March 16-23, 2013, in San Diego.

18 Mar 2013

Pivotal study of olesoxime in Spinal Muscular Atrophy receives positive interim review

Trophos SA, a clinical stage pharmaceutical company developing innovative therapeutics from discovery to clinical validation for under-served medical needs in neurology and cardiology, announced the completion of the interim analysis of the pivotal efficacy study of olesoxime in the rare neurodegenerative condition Spinal Muscular Atrophy (SMA).

1 Mar 2013

Systemic delivery of antisense oligonucleotides neutralizes mutant RNA toxicity in mice with DM1

By targeting the specific mutation that causes the hereditary neuromuscular disease myotonic dystrophy, it is possible to neutralize the mutant RNA toxicity and minimize or even eliminate the disabling symptoms of the disease.

25 Feb 2013

CUMC receives $7M to help establish Weinberg Family Cerebral Palsy Center

Seeking to bridge the transition from pediatric to adult care for people living with cerebral palsy, Debby and Peter A. Weinberg, with several of their family members and friends, have given more than $7 million to help establish the Weinberg Family Cerebral Palsy Center at Columbia University Medical Center (CUMC).

31 Jan 2013

Pfizer, Repligen sign licensing deal to advance spinal muscular atrophy program

Repligen Corporation announced today that it has entered into an exclusive worldwide licensing agreement with Pfizer Inc. to advance Repligen's spinal muscular atrophy (SMA) program, originally in-licensed from Families of SMA (FSMA).

3 Jan 2013

Leg press power best indicator of function after knee arthroplasty

Leg press power is a better indicator of performance-based and self-reported functionality after total knee arthroplasty than knee extension strength, show study findings.

23 Oct 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

Two CUMC studies provide new insights into spinal muscular atrophy

A study by scientists from the Motor Neuron Center at Columbia University Medical Center (CUMC) suggests that spinal muscular atrophy (SMA), a genetic neuromuscular disease in infants and children, results primarily from motor circuit dysfunction, not motor neuron or muscle cell dysfunction, as is commonly thought.

12 Oct 2012