Quest Magazine

Nonwalkers With SMA At High Risk for Weight Gain

Children and adults with types 2 and 3 spinal muscular atrophy (SMA) who are no longer walking have an increased risk of being overnourished and overweight if they otherwise have relatively good motor function, a multicenter study shows.

The results emphasize the importance of a "dedicated and experienced nutritionist" in SMA medical management, the researchers say.

About the study

Antisense Treatment Restores Full-Length SMN in SMA Mice

Scientists have found that mice with a disease resembling a mild form of spinal muscular atrophy (SMA) known as SMA type 3 showed more production of a needed protein in their spinal cords and more normal-looking ears and tails after treatment with a gene-modifying molecule that researchers hope could become a treatment for human SMA.

About the new findings

Disability Activist with SMA is a Globe Trotter

Life has been a whirlwind of activity for Victor Pineda lately.  The 31-year-old bachelor got married the first week in July.  He earns his Ph.D. in government planning around the first of August.

But these momentous events are almost insignificant compared to the rest of his typical schedule. In 2005 Pineda created the Victor Pineda Foundation (VPF) and today it commands most of his time.

Clothing Designers with MD: Striving for Sartorial Excellence

Four young men with powerful entrepreneurial spirits and a flair for design are making inroads in the country’s custom clothing industry. Although each of the three companies offers its own distinctive designs, the owner/operators have a lot in common.

Participants Sought for Three ALS Trials

Research scientists are seeking individuals interested in participating in any of the following three trials in ALS.

High-fat, high-calorie diets

Enrollment is open in an MDA-supported study comparing three tube-feeding formulas – one high in calories; one high in fat calories; and one standard formula – in people with ALS.

Lumizyme Now Commercially Available for Pompe

 Monique Griffin of Orlando, Fla., was one of the first in the nation to receive the commercially available treatment Lumizyme for acid maltase deficiency (AMD, or Pompe disease).

First US Exon-Skipping Trial Opens

Update (Nov. 5, 2012): Results announced in November 2012 showed the drug, now called drisapersen, reached blood levels approximately proportional to the injected dose at two of the three dosage levels tested and was not associated with serious adverse events. See Drisapersen Appears Safe in Non-Walking Boys With DMD.

It's Myasthenia Gravis Awareness Month

In recognition of June being National Myasthenia Gravis Awareness Month, Quest News Online is examining the disease from several perspectives. This article offers an overview of signs, symptoms, diagnosis and treatments for myasthenia gravis (MG), as well as some drugs for people with MG to avoid.

Augie's New Book Extols Giving

When MDA ALS Division Co-Chair Augie Nieto was a young businessman just starting out, he had an idea that laid the groundwork for his later, multimillion-dollar success in the fitness industry. Instead of selling his unwanted exercise bikes at a loss, he sent 50 of them as gifts to health club owners across the country. Soon, orders started pouring in.

The Flip of a Switch

Neuraltus Pharmaceuticals of Palo Alto, Calif., is developing a small molecule whose target is regulation of immune system cells believed to contribute to neuroinflammation and disease progression in ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease).

About ALS mechanisms  

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