A trial of the exon-skipping drug eteplirsen resulted in an increase in both dystrophin production and walking ability in boys with Duchenne MD
Update (Aug. 8, 2013): This story has been updated to reflect the Aug. 1, 2013, publication of these trial results in Annals of Neurology.
Boys with Duchenne MD who received 36 weekly infusions of an experimental exon-skipping drug showed a significantly slower decline in walking ability
Editor's note (July 30, 2012): This story was revised to include information about the specific mutations being targeted by eteplirsen.
Two companies will jointly develop a drug for type 1 myotonic dystrophy; UK survey responders say modafinil's benefits for MMD1-related sleepiness outweigh side effects
Type 1 myotonic dystrophy (MMD1, or DM1) and type 2 myotonic dystrophy (MMD2, or DM2) are complex, multisystem disorders caused by similar genetic flaws on chromosome 19 (MMD1) and chromosome 3 (MMD2). Treatments that target the underlying molecular causes of MMD1 and MMD2 are in development.
The complex and multifaceted disease known as myotonic muscular dystrophy (MMD) — also known as dystrophia myotonia (DM) — was the subject of an In Focus report in the April-June 2012 Quest.
Here, we delve into experimental strategies that may markedly improve the outlook for people with this disorder.
Experiments in SMA mice suggest that antisense treatment is most effective when it reaches all body tissues, not just the central nervous system
Treatment of a mouse model of severe spinal muscular atrophy (SMA) with an antisense oligonucleotide results in greater and longer-lasting benefit when given systemically than when given only to the central nervous system, new research shows.
Quest takes a look at gene therapy, 'antisense' and other cutting-edge scientific approaches and how they're being applied to diseases in MDA's program
Antisense oligonucleotides block flawed genetic instructions
Antisense oligonucleotides — also called antisense, oligos, or simply AONs — are pieces of genetic code that keep other genetic code from being processed. Designed to pair up with a particular sequence of DNA or RNA, AONs can change, block or destroy targeted genetic instructions in a variety of ways.
Investigators presented intriguing data from a trial of an experimental exon skipping drug for Duchenne muscular dystrophy, at the 2010 American Academy of Neurology meeting
The multinational pharmaceutical company GlaxoSmithKline (GSK) and the Dutch biotechnology company Prosensa announced "intriguing results" following a 12-person trial of an experimental "exon skipping" therapeutic for Duchenne muscular dystrophy (DMD).