High energy, optimism prevailed at a February 2014 congressional briefing for Capitol Hill staffers on reauthorizing key muscular dystrophy legislation
"The energy in the room was high, and the mood was optimistic," said Annie Kennedy, MDA's senior vice president of advocacy, speaking about the Congressional briefing on reauthorization of the MD-CARE Act that she attended and helped to organize. The briefing was co-hosted by MDA and other muscular dystrophy organizations in Washington, D.C., on Feb.
Ataluren (PTC124) for Duchenne-Becker MD related to premature stop codons did not receive approval in Europe prior to completion of an ongoing phase 3 trial
The experimental drug ataluren, in development for Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) caused by specific mutations in the gene for the dystrophin protein, will not receive conditional approval at this time from the European Medicines Agency (EMA). Ataluren's developer is requesting that the EMA re-examine the data.
Sarepta Therapeutics presented new favorable safety and efficacy results for its experimental exon-skipping drug eteplirsen, designed to treat Duchenne MD caused by specific dystrophin mutations
Update (Feb. 5, 2014): An analysis of pulmonary function tests at 120 weeks (2.3 years) found boys treated with eteplirsen showed a stabilization of respiratory function over a time period and in an age group where a decline would have been expected. Sarepta announced these results in a Feb. 5, 2014, press release.
An increase in dystrophin was seen in a foot muscle in 61 percent of boys with nonsense-mutation DMD treated with ataluren in an MDA-supported study
An increase in levels of the dystrophin protein was seen after treatment with the experimental drug ataluren (originally called PTC124) in an MDA-supported, phase 2a, open-label trial of 38 boys with Duchenne muscular dystrophy (DMD) caused by a specific type of mutation in the dystrophin gene.
Pharmaceutical company Eli Lilly is conducting a trial of tadalafil, which may help regulate blood flow to muscles, in some 300 boys with Duchenne MD
Multinational pharmaceutical company Eli Lilly is conducting a phase 3 trial of the drug tadalafil in approximately 300 boys with Duchenne muscular dystrophy (DMD) who are 7-14 years old, able to walk, have adequate cardiac function and meet other study criteria.
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
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
The National Institutes of Health is conducting a study to develop a questionnaire about motor function in young children who have a neuromuscular disorder