Muscular Atrophy News and Research

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Study shows how RNA splicing errors may trigger development of ALS, FTD

The most frequent genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD)- rare and related neurological disorders marked by progressive deterioration of motor or cognitive abilities- may be due to errors in RNA splicing, an intermediary step for translating genetic instructions into functional proteins.

14 Jun 2017

Study unravels new clues to cause and treatment of spinal muscular atrophy

Spinal muscular atrophy, a neurodegenerative disease that causes progressive muscle wasting and paralysis, may be partly due to abnormalities in the synapses that connect sensory neurons and motor neurons, according to researchers at Columbia University Medical Center.

16 May 2017

New gene variant linked to Lou Gehrig's disease

For the first time, a variant in UBQLN4 gene has been associated with Lou Gehrig's disease or amyotrophic lateral sclerosis (ALS) - a progressive disease resulting in the loss of nerve cells that control muscle movement, which eventually leads to paralysis and death.

15 May 2017

Researchers uncover new potential target for treating spinal muscular atrophy

Though spinal muscular atrophy in its most severe form remains incurable and fatal in early childhood, researchers are sustaining a multipronged counterattack for patients and their families. The first treatment for the disease gained U.S. market approval in December.

2 May 2017

Positive CHMP opinion recognizes efficacy of Biogen’s SPINRAZA for treatment of spinal muscular atrophy

Biogen today announced the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion recommending the granting of a marketing authorization for SPINRAZA to treat patients with spinal muscular atrophy (SMA).

21 Apr 2017

Targeted treatment slows progression of two degenerative brain diseases in mice

Scientists report a significant step toward combatting two degenerative brain diseases that chip away at an individual's ability to move, and think.

12 Apr 2017

Researchers use mathematical modeling to raise density of bones badly degraded by osteoporosis

They may seem rigid and set in their ways, but your bones are actually under constant construction and deconstruction. They give up their nutrient treasures (calcium) to the body and then rebuild in a constant give-and-take sort of rhythm.

29 Mar 2017

Emory researchers reveal 'matchmaker' role for protein linked to SMA

A puzzling question has lurked behind SMA, the leading genetic cause of death in infants. The disorder leads to reduced levels of the SMN protein, which is thought to be involved in processing RNA, something that occurs in every cell in the body.

14 Feb 2017

Designer compound may be effective tool for tackling tau-related disorders

In a study of mice and monkeys, National Institutes of Health funded researchers showed that they could prevent and reverse some of the brain injury caused by the toxic form of a protein called tau.

8 Feb 2017

Synthetic molecule appears to reverse Alzheimer's-related neurological damage

Under ordinary circumstances, the protein tau contributes to the normal, healthy functioning of brain neurons. In some people, though, it collects into toxic tangles that damage brain cells.

25 Jan 2017

Orphan Disease Center establishes new initiative that focuses on ALS

The Orphan Disease Center in the Perelman School of Medicine at the University of Pennsylvania has established a new Program of Excellence for Motor Neuron Disease.

10 Jan 2017

Johns Hopkins researchers identify new biological target for treating spinal muscular atrophy

Johns Hopkins researchers along with academic and drug industry investigators say they have identified a new biological target for treating spinal muscular atrophy.

9 Jan 2017

CSHL researcher-inventor hails FDA approval of new SMA drug

Within a week of Christmas day, a drug called nusinersen will be in the hands of doctors across the nation, who will use it, most urgently, to treat young children with a severe and potentially fatal illness called spinal muscular atrophy (SMA).

26 Dec 2016

Ionis announces FDA approval of first SMA drug in the U.S for pediatric and adult patients

Ionis Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration has approved SPINRAZATM (nusinersen) under Priority Review for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

26 Dec 2016

MDA celebrates FDA approval of new spinal muscular atrophy drug

The Muscular Dystrophy Association today celebrated news of the U.S. Food and Drug Administration's decision to grant approval for nusinersen (brand name Spinraza), the first disease-modifying drug to treat the most common genetic cause of death in infants.

23 Dec 2016