Quest Magazine

SMA: Masking Unwanted Instructions

Scientists at three U.S. institutions have used a very small synthetic molecule to correct the genetic defect in cells taken from a person with spinal muscular atrophy (SMA), a disease in which muscle-controlling nerve cells in the spinal cord are lost.

Iplex on Hold

The Richmond, Va., biopharmaceutical company Insmed announced July 27, 2009, that it will not supply its experimental drug Iplex to any new patients with amyotrophic lateral sclerosis (ALS) for the foreseeable future, and that it intends to analyze the available data on Iplex for ALS and type 1 myotonic dystrophy (MMD1, or DM1) before deciding whether to proceed with development of the drug for either dise

Iplex on Hold

The Richmond, Va., biopharmaceutical company Insmed announced July 27, 2009, that it will not supply its experimental drug Iplex to any new patients with amyotrophic lateral sclerosis (ALS) for the foreseeable future, and that it intends to analyze the available data on Iplex for ALS and type 1 myotonic dystrophy (MMD1, or DM1)

ALS TDI Update

On July 16, 2009, the MDA-supported ALS Therapy Development Institute (ALS TDI) gave its quarterly research update in a Webcast that's now archived on the Institute's site and accessible to all who register. (See Research Update Q209.)

ALS TDI Update

On July 16, 2009, the MDA-supported ALS Therapy Development Institute (ALS TDI) gave its quarterly research update in a Webcast that's now archived on the Institute's site and accessible to all who register. (See Research Update Q209.)

Through the Pipeline

On July 16, 2009, the MDA-supported ALS Therapy Development Institute (ALS TDI) gave its quarterly research update in a Webcast that's now archived on the Institute's site and accessible to all who register. (See Research Update Q209.)

Iplex on Hold

Jury Still Out on Value of Vitamin C in CMT1A

A recent trial has shown inconclusive results from various doses of vitamin C (ascorbic acid) in patients with the peripheral nerve disease type 1A Charcot-Marie-Tooth disease (CMT1A), and the jury remains out on this form of treatment, says Michael Shy, an MDA grantee at Wayne State University in Detroit.

ALS: Riding for Augie

Many fundraising events for Augie’s Quest (MDA’s aggressive ALS research initiative) tend to be glamorous galas attracting high-profile socialites to genteel settings.

There was nothing genteel about the fundraiser that began June 20 as part of Race Across America (RAAM). It’s one of the toughest endurance events in the world. Cyclists – usually 250 or so, solo or in teams – race 3,000 miles from Oceanside, Calif., to Annapolis, Md. Four-person teams typically cover that distance in five to eight days of nightmarish physical challenge.

Genetics: You, Me and PGD

Update 5/21/12:This study is closed, and results are available at Decision Making About PGD Is Complex, Study Finds. Information about future studies will be posted at PGD: Couples' Decision Making when available.

Preimplantation genetic diagnosis (PGD) is a way for couples to avoid passing along a hereditary disease to their offspring by pre-screening embryos.

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