Quest Magazine

NIH Convenes Meeting on SMA Treatment Development

In a sign of the significant progress being made in spinal muscular atrophy (SMA) research, the National Institutes of Health (NIH) convened a meeting in October that examined the status of laboratory research, clinical trials and strategies for drug development in this disease, with an eye toward speeding the process.

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.

Power Soccer Champ with SMA Excels on Multiple Fronts

Ben Carpenter, a young man with many talents.

Don’t be surprised if, in a few years, you hear about some fascinating new amusement park rides. And don’t be surprised if a young man named Ben Carpenter proves to be the genius behind the design of those rides.

Encouraging Results in LGMD Gene Therapy Trial

Results from an MDA-supported, phase 1 study of gene therapy for the type 2D form of limb-girdle muscular dystrophy (LGMD2D) show sustained protein production from the transferred genes in two out of three trial participants six months after injection of the genes into a foot muscle.

The new results follow an announcement last year showing protein production from transferred genes in the first three participants in this trial at either six weeks or three months after the gene transfer.

Early-Life Protein, Made Too Late, Causes Trouble in FSHD

Little by little, the molecular underpinnings of facioscapulohumeral muscular dystrophy (FSHD) are yielding to scientific investigations. The latest revelations about a protein known as DUX4, announced in October, could bring a treatment for FSHD closer to the clinic.

About recent FSHD research

Viagra May be Heart Helper in DMD

Results from a recent study have shown that treatment with the drug sildenafil (brand name Viagra) conferred both long-term protection against cardiac (heart) dysfunction in younger mice, and rapid reversal of heart damage in aged mice with a disease resembling Duchenne muscular dystrophy (DMD).

New Apprenticeship Program for Personal Care Attendants

A new training opportunity, the Direct Support Professional Registered Apprenticeship Program, will assist direct support professionals (such as hired caregivers and aides) in advancing in this essential field, say the program’s creators, the National Alliance for Direct Support Professionals (NADSP) and the American Network of Community Options and Resources (ANCOR).

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

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