Quest Magazine

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.)

Two-Drug Combo Makes a Difference in FA

Results from a clinical trial conducted in Spain involving 20 individuals with Friedreich's ataxia (FA) show that a combination therapy using two drugs, idebenone and deferiprone (DFP), demonstrated a "stabilizing effect" in neurological function and reduced abnormal heart-muscle enlargement in participants who received the two-drug combo for a period of 11 months.

Research Briefs: DMD

Anti-myostatin drug trial shows good preliminary results

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.

Misfolded SOD1 Found in Sporadic ALS Samples

A new study suggests misfolding of the SOD1 protein may be a common contributor not only to familial (inherited) amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene but to the more common sporadic (noninherited) form of the disease as well.

Intravenous AVI4658 Shows Safety, Benefit in DMD

The experimental drug AVI4658, in development by AVI BioPharma to treat Duchenne muscular dystrophy (DMD) caused by specific genetic mutations, was well tolerated and resulted in increased production of the needed dystrophin protein. Measured aspects of cardiac, pulmonary and skeletal muscle function remained stable.

Low-Dose Ataluren Shows Some Benefit in DMD/BMD

A low-dose regimen of ataluren (formerly called PTC124), an experimental drug developed by PTC Therapeutics to treat  Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) caused by a certain type of genetic mutation, is superior to a high-dose regimen or a placebo.

ALS TDI Conference Summarizes ALS Research Progress

Themes discussed throughout the research symposium included ALS genetics, clinical trials, the role of the immune system in disease onset and progression, biomarkers, and the use of stem cells in ALS therapeutics.

Diaphragm Pacing System Awaiting FDA Approval for ALS

The Synapse Biomedical NeuRx Diaphragm Pacing System (DPS), a device that stimulates the respiratory diaphragm with electrical signals, has received Humanitarian Use Device designation from the U.S. Food and Drug Administration (FDA), allowing it to be used by people with amyotrophic lateral sclerosis (ALS) who have a “stimulatable” diaphragm and are experiencing chronic breathing problems.

Comic with BMD Builds Accessibility One Doorway at a Time

Jonah Bascle (rhymes with “rascal”) unleashes his zany brand of stand-up comedy on New Orleans nightclub and restaurant patrons four or five times a week. As an unusual consequence of these gigs, more and more of the historical city’s old buildings are becoming accessible to people in wheelchairs.

As a guy who uses a wheelchair himself due to Becker muscular dystrophy, Bascle, 24, knows first hand how difficult it is to navigate the entrances to many long-established buildings in the legendary home of Mardi Gras.

Pages