DMD/BMD: Stop Codon Read-Through Drug Trial Still Recruiting Participants

PTC Therapeutics' phase 3 trial of its experimental drug ataluren remains open to boys with Duchenne or Becker MD caused by premature stop codon mutations

Production of full-length, functional protein molecules is the goal of stop codon read-through drugs like ataluren.
by Margaret Wahl on December 9, 2013 - 9:37am

PTC Therapeutics, a South Plainfield, N.J., biopharmaceutical company, wants to remind families affected by Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) caused by specific genetic mutations that its phase 3 trial of ataluren is recruiting patients at 53 sites in the U.S., Europe, and several other countries.

Ataluren — sometimes referred to as a stop codon read-through drug — is an experimental compound that targets a type of genetic mutation affecting approximately 10 to 15 percent of boys with DMD or BMD.

The type of mutation, called a premature stop codon mutation or nonsense mutation, causes cells to stop making a protein before the process is complete, resulting in synthesis of a nonfunctional or partially functional protein.

Stop codon read-through for DMD or BMD is an experimental strategy that coaxes cells to "read through" (ignore) a premature stop signal in the genetic instructions for the dystrophin protein, so that full-length, functional dystrophin protein can be made in muscle cells.

MDA has supported development of ataluren (originally called PTC124) through a 2005 grant to PTC for $1.5 million.

To help families understand the ataluren trial, PTC has posted a Frequently Asked Questions section on its website.

Additional questions can be addressed to Diane Goetz, senior director of patient and professional advocacy at PTC. Call the toll-free number (866) 282-5873 or send an email to patientinfo@ptcbio.com.

For study details and contact information for the study sites, see Phase 3 Study of Ataluren in Patients with Nonsense Mutation Duchenne Muscular Dystrophy, or enter NCT01826487 in the search box at ClinicalTrials.gov.

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