Ezutromid Shows Potential in DMD | Medpage Today

Ezutromid Shows Potential in DMD | Medpage Today: "LOS ANGELES -- The investigative utrophin modulator ezutromid showed promise in treating Duchenne muscular dystrophy, according to a interim analysis of an open-label, phase II clinical trial.

Ezutromid appeared to have significantly deceased inflammation in the calf muscle of Duchenne patients after 24 weeks, reported Anne Heatherington, PhD, of Summit Therapeutics in Oxford, England, and colleagues at the American Academy of Neurology annual meeting."



'via Blog this'

Research establishes use of chimeric cells as potential therapy for Duchenne muscular dystrophy

Research establishes use of chimeric cells as potential therapy for Duchenne muscular dystrophy: "Cells made by fusing a normal human muscle cell with a muscle cell from a person with Duchenne muscular dystrophy --a rare but fatal form of muscular dystrophy -- were able to significantly improve muscle function when implanted into the muscles of a mouse model of the disease. The findings are reported by researchers from the University of Illinois at Chicago in the Stem Cell Review and Reports."



'via Blog this'

Summit’s DMD drug cuts muscle inflammation in POC trial | FierceBiotech

Summit’s DMD drug cuts muscle inflammation in POC trial | FierceBiotech: "Summit Therapeutics has posted further data from the ongoing phase 2 trial of ezutromid. The latest analysis links the utrophin modulator to a statistically significant decline in muscle inflammation in boys with Duchenne muscular dystrophy (DMD).

Oxford, U.K.-based Summit generated the data on muscle inflammation by measuring MRI transverse relaxation time T2 (MRS-T2). The technique quantifies the breakdown and inflammation of muscle, making it a useful tool for assessing the progression of DMD. MRS-T2 values rise as DMD progresses but can be brought down by steroids.

The Summit data suggest ezutromid may drive reductions in MRS-T2 beyond those achieved by the use of steroids alone. All 38 participants in the proof-of-concept trial were on stable steroid regimes. After 24 weeks of twice-daily ezutromid doses, MRS-T2 decreased by 0.861 milliseconds, on average."



'via Blog this'

Cell therapy for heart disease caused by muscular dystrophy also improves limb strength, study shows

Cell therapy for heart disease caused by muscular dystrophy also improves limb strength, study shows: "Injections of cardiac progenitor cells help reverse the fatal heart disease caused by Duchenne muscular dystrophy and also lead to improved limb strength and movement ability, a new study shows.

The study, published today in Stem Cell Reports, showed that when researchers injected cardiosphere-derived cells (CDCs) into the hearts of laboratory mice with muscular dystrophy, heart function improved along with a marked increase in exercise capacity.

"We unexpectedly found that treating the heart made the whole body better," said Eduardo Marbán, MD, PhD, director of the Smidt Heart Institute and the investigator who developed the cardiosphere-derived cell technology used in the study. "These basic findings, which have already been translated to clinical trials, rationalize why treating the heart may also benefit skeletal muscle function in boys and young men with Duchenne.""



'via Blog this'

Robotic arm developed for Duchenne patients -- ScienceDaily

Robotic arm developed for Duchenne patients -- ScienceDaily: "Researchers from the University of Twente's MIRA research centre, together with the VUmc, TU Delft and the Radboud umc, have developed the A-Gear: a robotic arm that can support the daily activities of people suffering the muscular disease Duchenne Muscular Dystrophy. They recently put the final touches to the first prototype for a discreet, body-connected support aid that can be worn under the clothing and that can support independent operation of the arm during important daily activities."



'via Blog this'

New CRISPR gene-editing technique can correct majority of mutations that cause DMD

New CRISPR gene-editing technique can correct majority of mutations that cause DMD: "Scientists have developed a CRISPR gene-editing technique that can potentially correct a majority of the 3,000 mutations that cause Duchenne muscular dystrophy (DMD) by making a single cut at strategic points along the patient's DNA, according to a study from UT Southwestern Medical Center.

The method, successfully tested in heart muscle cells from patients, offers an efficient alternative to the daunting task of developing an individualized molecular treatment for each gene mutation that causes DMD. It also opens up possible new treatment approaches for other diseases that have thus far required more intrusive methods to correct single-gene mutations.

Scientists say the new strategy enhances the accuracy for surgical-like editing of the human genome, correcting mistakes in the DNA sequence that cause devastating diseases like DMD, a deadly condition caused by defects in the dystrophin gene. Normally, the dystrophin protein helps strengthen muscle fibers."



'via Blog this'

Ezutromid Reduced Muscle Damage in DMD Patients, Interim Data Shows

Ezutromid Reduced Muscle Damage in DMD Patients, Interim Data Shows: "At 24-weeks, muscle damage and utrophin protein were measured through muscle biopsies. Muscle biopsies were taken before treatment, and 24 patients had their second biopsy after 24 weeks of treatment. The remaining patients will have their second one by the end of the study (48 weeks).

Muscle damage was significantly reduced after 24 weeks, based on a 23% decrease in the levels of developmental myosin, compared with baseline. The decrease of developmental myosin was observed in about 64% of patients (14 patients), with five of them showing more than 40% reduction."



'via Blog this'