Muscular Atrophy News and Research

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Urine exRNA may be source of biomarkers for muscular dystrophy

Massachusetts General Hospital researchers have found that extracellular RNA in urine may be a source of biomarkers for the two most common forms of muscular dystrophy, noninvasively providing information about whether therapeutic drugs are having the desired effects on a molecular level.

26 Sep 2018

Biogen and Eisai announce results of LTE Phase 1b study of aducanumab for treating MCI

Biogen and Eisai Co., Ltd. announced results from a recent analysis of the ongoing long-term extension Phase 1b study of aducanumab, an investigational treatment for mild cognitive impairment due to Alzheimer's disease and mild AD dementia.

24 Sep 2018

Study links spinal muscular atrophy disease expression with haplotypes

A natural history study has provided the first comprehensive clinical description of spinal muscular atrophy within the Amish and Mennonite communities and correlates ancestral chromosome 5 haplotypes and SMN2 copy number with disease severity.

7 Sep 2018

SMA treatment may be effective even if started in children seven months and older

A drug shown to be effective in the treatment of babies with the rare muscle-wasting disease spinal muscular atrophy may be effective for muscle control even when treatment is started in children seven months and older, according to a study published in the August 29, 2018, online issue of Neurology, the medical journal of the American Academy of Neurology. Previous studies focused on children younger than seven months old.

30 Aug 2018

Alternative method identified for delivering nusinersen to spinal muscular atrophy patients

A new report has identified an alternative method to deliver nusinersen to patients with spinal muscular atrophy using a subcutaneous intrathecal catheter system configured by connecting an intrathecal catheter to an implantable infusion port.

24 Aug 2018

MDA announces 34 new grants totaling $9.9 million to accelerate neuromuscular research

The Muscular Dystrophy Association today announced the award of 34 new grants totaling more than $9.9 million for its summer round of funding. These new grants represent a continued commitment by MDA to fund groundbreaking research that will accelerate treatments and cures for the more than 40 diseases in its program.

24 Aug 2018

Study findings highlight importance of early detection of SMA through newborn screening

Spinal muscular atrophy is a genetic disease that affects motor neurons in the spinal cord, resulting in muscle atrophy and widespread weakness that eventually impair swallowing and breathing.

17 Jul 2018

Novel therapeutic approach alleviates ALS-related muscular atrophy in mouse models

Supplementing a single protein found in the spinal cord could help prevent symptoms of Lou Gehrig's disease, according to a new study out of Case Western Reserve University School of Medicine.

12 Jul 2018

Researchers find novel ways to improve participation in clinical research

Videos and creative uses of other visuals provide a novel way to obtain informed consent during clinical trials to improve participants' understanding and retention of trial information, according to a study by Nemours Children's Health System presented at the American Thoracic Society (ATS) Annual Conference.

25 May 2018

Scientists discover how potential new drug targets spinal muscular atrophy

Spinal muscular atrophy (SMA) is a genetic disease that can leave infants with weak muscles and trouble breathing. Many with the disease die before age two. To help these patients, doctors need therapies that target the genetic mutation and stop its progression.

8 May 2018

Therapeutic RNA corrects error in genetic processing that leads to familial dysautonomia

Scientists at Cold Spring Harbor Laboratory have identified a therapeutic RNA molecule that corrects the error in genetic processing that leads to familial dysautonomia, a rare inherited neurodegenerative disorder.

30 Apr 2018

Investigational drug may help increase protein levels in babies with spinal muscular atrophy

A preliminary study suggests that an investigational drug may help increase protein levels in babies with spinal muscular atrophy. The open-label study is released today and will be presented at the American Academy of Neurology's 70th Annual Meeting in Los Angeles, April 21 to 27, 2018.

19 Apr 2018

Scientists discover basic molecular mechanism that helps understand how ALS works

It took eight long years of research, but now an international team led by neuroscientists at Université de Montréal has discovered a basic molecular mechanism that better helps understand how Lou Gehrig's disease, or amyotrophic lateral sclerosis (ALS), works.

19 Mar 2018

Impaired transport processes in neurons contribute to neurodegenerative disorders

Two partners have to find their way to each other in order to transport substances to the remote ends of special nerve fibers to maintain their function. If this partnering process is disturbed, the cells may die in the worst case, resulting in neurodegenerative disorders such as amyotrophic lateral sclerosis (ALS) or spinal muscular atrophy.

9 Mar 2018

Researchers develop method to identify splicing-based biomarkers for liver cancer

Because liver cancer is particularly diverse, genetically, and prone to relapse, identifying biomarkers that can predict disease progression is a critical goal in the fight against it.

2 Mar 2018

New medication improves motor function in children with later-onset spinal muscular atrophy

Children with later-onset spinal muscular atrophy (SMA) were more likely to show gains in motor function when treated with a new medication compared to children receiving a sham procedure, according to a study published today in the New England Journal of Medicine.

15 Feb 2018

Muscular Atrophy News and Research

Urine exRNA may be source of biomarkers for muscular dystrophy

Biogen and Eisai announce results of LTE Phase 1b study of aducanumab for treating MCI

Study links spinal muscular atrophy disease expression with haplotypes

SMA treatment may be effective even if started in children seven months and older

Alternative method identified for delivering nusinersen to spinal muscular atrophy patients

MDA announces 34 new grants totaling $9.9 million to accelerate neuromuscular research

Study findings highlight importance of early detection of SMA through newborn screening

Novel therapeutic approach alleviates ALS-related muscular atrophy in mouse models

Researchers find novel ways to improve participation in clinical research

Scientists discover how potential new drug targets spinal muscular atrophy

Therapeutic RNA corrects error in genetic processing that leads to familial dysautonomia

Investigational drug may help increase protein levels in babies with spinal muscular atrophy

Scientists discover basic molecular mechanism that helps understand how ALS works

Impaired transport processes in neurons contribute to neurodegenerative disorders

Researchers develop method to identify splicing-based biomarkers for liver cancer

New medication improves motor function in children with later-onset spinal muscular atrophy

Scientists uncover the structure of microtubule motor proteins

UNC gene therapy scientists uncover viral barcode to penetrate blood-brain barrier

Researchers discover gene network that regulates the motor neuron development in growing embryo

Researchers describe severe toxicity with AAV9-based high-dose gene therapy in animal models

Industry Focus eBook - Analytical Chemistry (2nd Edition) eBook

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