Amyotrophic Lateral Sclerosis (ALS)

VCP Gene Implicated in Familial ALS, IBM

A multinational study group, using cutting-edge "exome sequencing" technology, has uncovered five mutations in the valosin-containing protein (VCP) gene and implicated them as molecular causes of some familial forms of ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease).

ALS Experts Gather to Exchange Ideas

New insights and perspectives on the biology of amyotrophic lateral sclerosis (ALS), best practices in the care of individuals with the disease, and future directions for development of ALS therapies were the central themes of the 21st International Symposium on ALS/MND (Motor Neuron Disease) that took place in Orlando, Fla., Dec. 11-13, 2010.

ALS Research Briefs

Immune system regulator shows safety in ALS

Neuraltus Pharmaceuticals announced Nov. 30, 2010, that its experimental drug NP001 was safe and well-tolerated at four different dose levels when given as a single intravenous treatment to people with ALS. There was also a statistically significant improvement in blood levels of a biological marker (biomarker) thought to be involved in ALS disease progression.

'New and Important Player' Advances Understanding of ALS

A population of stem cells called NG2+ cells — which mature into central nervous system support cells called oligodendrocytes — exhibit significant developmental differences in healthy mice compared to mice with a disease resembling human ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease).

What's a 'Meaningful' Change for Those with ALS?

What's a meaningful change for someone with amyotrophic lateral sclerosis (ALS) or his or her caregiver? A new, MDA-supported study seeks to answer that question.

It's believed that physical or psychosocial changes associated with a new treatment that may be "statistically significant" when clinical trial results are analyzed may not be the same as changes that people with ALS and their caregivers consider meaningful.

‘Simple Pleasures’ Essay by Writer with ALS Published in NYT

Neil Selinger, 57, of Larchmont, N.Y., was a high-powered attorney working on high-profile, high-dollar cases when he decided to set it all aside in 2007 to pursue volunteering and writing. 

In 2009, his life underwent yet another change when he received a diagnosis of ALS. The disease hasn’t stopped Selinger’s writing, but it has added a new slant to his point of view.

Does Spirituality Influence Acceptance of Disability?

With approximately 54 million Americans living with disabilities, exploring and understanding factors that might facilitate or hinder acceptance of one’s disability may be an important area of research.

But what exactly is the relationship between religious/spiritual attitudes and acceptance or lack of acceptance of disability for people with neuromuscular disorders?

Emotional Expression Medication Approved for Use in ALS

The U.S. Food and Drug Administration (FDA) has approved Nuedexta for "pseudobulbar affect" (PBA), a neurologic condition that sometimes occurs in people with amyotrophic lateral sclerosis (ALS) and involves episodes of involuntary emotional expression out of proportion to or distinct from the person's actual emotional state. The approval was announced by Nuedexta's developer, Avanir Pharmaceuticals, Oct. 29, 2010.

ALS Stem Cell Trial is Moving Forward

The Maryland biotherapeutics company Neuralstem will move its clinical trial of neural stem cells in people with amyotrophic lateral sclerosis (ALS) into its next stage, following a thumbs-up from the trial's safety monitoring board, the company announced Oct. 18, 2010. (See Neuralstem Updates ALS Clinial Trial Progress.)

National ALS Registry Now Open

After two years of laying the groundwork, the Agency for Toxic Substances and Disease Registry (ATSDR) has opened its National ALS Registry to collect information from people living with ALS.

Scientists at the ATSDR, which is part of the Centers for Disease Control and Prevention in Atlanta, hope registry data will provide insight into the causes of, and potential treatments for, ALS.