Preliminary results from four infants in a phase 2 trial of ISIS-SMNRx suggest the drug is well-tolerated and may prolong ventilator-free survival
Interim results of a phase 2 clinical trial to test multiple doses of the experimental drug ISIS-SMNRx in infants with spinal muscular atrophy (SMA) suggest that the drug is well-tolerated and may prolong ventilator-free survival.
An MDA-supported team has developed mice with transplanted muscle tissue from people with facioscapulohumeral MD that is likely to speed research
Researchers funded in part by MDA have created a new research mouse (mouse "model") that they say is likely to speed investigations in facioscapulohumeral muscular dystrophy (FSHD) and therapy development for this disease. It's expected to complement an existing FSHD mouse model.
A research study of the use of the CoughAssist machine is open to children and teens with Ullrich congenital muscular dystrophy or Bethlem myopathy
Children and adolescents with Ullrich congenital muscular dystrophy or Bethlem myopathy are invited to participate in a study of a daily breathing treatment regimen using a brand-name device called a CoughAssist.
MDA-funded researchers say SMN, which is deficient in spinal muscular atrophy, is most needed during early life and in skeletal muscle and nerve tissues
Raising levels of the SMN protein, which is deficient in patients with the most common form of spinal muscular atrophy (SMA), has been the holy grail of therapy for this disease since the 1990s and is the goal of several experimental compounds now in development.
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.
MDA-supported researchers have developed compounds that block harmful interactions between a needed protein and abnormal RNA in cells with a type 2 myotonic dystrophy defect
MDA-supported investigators have designed two new compounds that precisely target the molecular defect that underlies type 2 myotonic muscular dystrophy (MMD2, or DM2), based on atomic-level imaging studies of the defect, also conducted in their laboratory.
BioMarin has purchased the rights to Repligen’s histone deacetylase (HDAC) inhibitor portfolio, including the experimental FA drug RG2833, whose development MDA has supported
Experimental Friedreich's ataxia (FA) drug RG2833, which has been in early-stage development by Massachusetts company Repligen with support from MDA, is now owned by California biotechnology company BioMarin.