Britain is now the first country in the world that allows the fusion of DNA from three people for the creation of babies. This could provide a solution for preventing diseases such as muscular dystropy, a hereditary disease characterized by progressive weakening of the muscles. The decision involved a change to in vitro fertilization…
News
Parent Project Muscular Dystrophy (PPMD), a nonprofit organization fighting against Duchenne muscular dystrophy (Duchenne) recently designated the University of Iowa Children’s Hospital (UI Children’s Hospital) a Certified Duchenne Care Center. UI Children’s Hospital is now the 7th center certified by PPMD, and is recognized for its dedication to improving care…
News
Ongoing Clinical Trial Testing New Method for Monitoring Duchenne Muscular Dystrophy Progression
A new pilot clinical trial on Duchenne Muscular Dystrophy, entitled “Double Push Acoustic Radiation Force (DP ARF) Ultrasound for Monitoring Muscle Degeneration in Duchenne Muscular Dystrophy,” is now enrolling participants. The trial is a prospective observational study to test the capacity of a new ultrasound-based imaging…
The Muscular Dystrophy Association (MDA) and Lowe’s stores are joining efforts to launch the MDA’s Shamrock program, the largest St. Patrick’s Day philanthropic event dedicated to help patients who live with the disabling muscle disease Duchenne muscular dystrophy. The FORTUNE 100 home improvement…
A recent study was published in The Lancet Neurology journal showing that a specific cardiac drug is able to induce cardiac function improvement in boys with muscular dystrophy and cardiomyopathy. The study is entitled “Eplerenone for early cardiomyopathy in Duchenne muscular dystrophy: a…
BioBlast Pharma Ltd. presented the preliminary results from its HOPEMD Phase 2/3 study investigating Cabaletta (trehalose) in patients with Occulopharyngeal Muscular Dystrophy (OPMD), a progressive, incapacitating genetic disease. Cabaletta is a chemical chaperone that has been shown to reduce pathological aggregation of proteins within cells in several diseases…
Dr. Thomas Gionis The use of stem cell therapy for a wide range of diseases continues to gain momentum across the United States, with more and more patients and their families seeking information about the benefits and potential risks of the treatment. In answer to this, clinics such as…
The Muscular Dystrophy Association (MDA) has released a report on the latest findings and developments on treatments for Duchenne muscular dystrophy that combat fibrosis and heart conditions associated with the disease. The Drug Development for DMD: February 2015…
A new study entitled, “Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers” was recently completed through a joint collaboration between researchers from the CNRS, UVSQ and INSERM at both the Laboratoire END-ICAP and the University of Bern. They discovered that through RNA “surgery,” synthetic oligonucleotides may…
News
tcDNA-antisense Oligonucleotides Offer Promising, New Therapeutics for Duchenne Muscular Dystrophy
A new study entitled “Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers” shows a new class of antisense oligonucleotides – tcDNA- antisense oligonucleotides – are highly efficient at rescuing dystrophin levels and improving symptoms in a mouse model of Duchenne muscular dystrophy.
Recent Posts
- In our new home, we will have more space for our sons with DMD
- MDA Engage: Seminar connects neuromuscular community in Hershey
- Our short film, ‘Lov-Able,’ heads to Australian disability-led film festival
- FDA set to decide on experimental DMD treatment in early 2027
- I wanted my oldest son with DMD to go to college more than he did