Muscular Atrophy News and Research

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New vaccine shows promise against toughest HIV strains

A new study reports successful immunization against a so-far invincible “death star” strain of an HIV-related laboratory virus (SIVmac239) and another tough strain. The new approach also achieves long-term protection with a single dose, showing that gene therapy could allow broad and durable protection, based on a modified form of the CD4 protein that allows HIV entry into human cells.

24 Jul 2019

BIA Separations selected to support AveXis’ gene therapy pipeline

BIA Separations, a leading biochromatography development and manufacturing company, today announced it is collaborating with AveXis, a Novartis company, to enhance the commercial purification process for its gene therapy pipeline, beginning with Zolgensma (onasemnogene abeparvovec-xioi), the first U.S. Food and Drug Administration-approved gene therapy indicated for the treatment of pediatric patients with spinal muscular atrophy (SMA) who are less than two years of age.

22 Jul 2019

Newly approved gene therapy for SMA is based on delivery vehicle discovered by Penn expert

James Wilson, MD, PhD, recalls being struck by the devastating toll of rare diseases as a young physician in the 1980s. He set out on a path to correct the genes that cause these conditions, including spinal muscle atrophy (SMA), the most common inherited fatal disease in infants.

25 May 2019

Quanterix’ Simoa technology powers more than 85 percent of Neurofilament light biomarker research to be unveiled at American Academy of Neurology Annual Meeting

Quanterix announce that its ultra-sensitive Simoa technology is the driving force behind 36 of the 42 Nf-L abstracts being presented at AANA.

From Quanterix 6 May 2019

$150,000 grant will support research on spinal muscular atrophy

EL PASO, Texas-Cure SMA recently awarded a $150,000 research grant to Laxman Gangwani, Ph.D., associate professor of Molecular and Translational Medicine, Center of Emphasis in Neurosciences, at TTUHSC El Paso's Paul L. Foster School of Medicine.

2 May 2019

Unique gene therapy approach paves new way to tackle rare, inherited diseases

Nonsense mutations are single-letter errors in the genetic code that prematurely halt the production of critical proteins. These unfinished proteins are unable to function normally, and nonsense mutations cause 10-15 percent of all inherited genetic diseases, including Duchenne muscular dystrophy, spinal muscular atrophy, cystic fibrosis and polycystic kidney disease.

19 Feb 2019

New form of therapy may stop or reverse progressive vision loss

A new form of therapy may halt or even reverse a form of progressive vision loss that, until now, has inevitably led to blindness.

1 Nov 2018

Biogen and Eisai present detailed results from phase 1b study of aducanumab at CTAD meeting

Biogen and Eisai Co., Ltd. announced that Biogen presented results at the Clinical Trials on Alzheimer's Disease meeting, in Barcelona, Spain, from the recent 36- and 48-month analyses of the ongoing long-term extension of the Phase 1b study of aducanumab, an investigational treatment for mild cognitive impairment due to Alzheimer's disease and mild AD.

29 Oct 2018

Novartis: Lancet publishes pioneering study of Aimovig’s efficacy in episodic migraine patients

Novartis announced today that the full data from the LIBERTY study of Aimovig (erenumab) in episodic migraine patients who had tried and failed two to four prior preventive treatments have been published in the Lancet.

23 Oct 2018

Genentech announces clinical data from studies investigating risdiplam in spinal muscular atrophy

Genentech, a member of the Roche Group, announced today interim clinical data from the dose-finding parts of the pivotal FIREFISH and SUNFISH studies investigating risdiplam in spinal muscular atrophy.

3 Oct 2018

Researchers discover how nerve cells control movement

Researchers at Karolinska Institutet in Sweden have discovered a new way in which nerve cells can control movement. In a study on zebrafish published in the journal PNAS they show that the contact between neurons and muscles is more dynamic than previously thought.

3 Oct 2018

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