Neuromuscular

The pediatric critical care medicine attending physician in the Department of Anesthesiology and Critical Care Medicine at the Children’s Hospital of Philadelphia spoke about how physicians can better address brain death in pediatric patients.

Neurology News Network for the week of February 23, 2019.

Over the past several years, scientific teams have developed investigational methods for delivering a gene to correct a mutation in the DMD gene which causes DMD by creating dysfunction in a patient’s dystrophin production.

The professor of neurology and pediatrics at Columbia University Irving Medical Center spoke about the success of the small molecule in older infants with SMA, and about what she’s looking forward to in the future of SMA treatment.

The investigational drug candidate, formerly known as CK-2127107, is the subject of the phase 2b FORTITUDE-ALS trial, which is assessing the effect of reldesemtiv compared with placebo on respiratory function and additional measures of skeletal muscle function.

The professor of neurology and pediatrics at Columbia University Irving Medical Center spoke about the treatment’s success thus far and its potential to treat infants with more severe disease.

The framework consists of 2 documents that expand on the agency’s plans for its risk-based approach for describing drugs, devices, and biologics, including those designated as regenerative medicine advanced therapies.

The professor in the Department of Neurology at Klinikum Wels-Grieskirchen, in Wels, Austria, spoke about the anti-CD20 antibody and its potential to treat patients with MG.

The therapy, which has a PDFUA date of Aug. 19, 2019, is being studied in an ongoing phase 3 study.

The chief medical officer of Cytokinetics spoke about the promise that the investigational fast skeletal muscle troponin activator holds as a potential treatment for spinal muscular atrophy.

Neurology News Network for the week of February 14, 2019.

At the final follow-up at an average of 20 months, remission was present in 42.9% of the 56 patients included, and an additional 25% had minimal manifestations or better as measured by the Myasthenia Gravis Foundation of America-Postintervention Status.

The director of the mitochondrial research program at Akron Children’s Hospital spoke about how to best manage a condition such as PMM, where there are no FDA-approved therapies.

The study provided Class I evidence that the ALSDI can help the confirmation and diagnosis of ALS and its mimicking conditions at the earliest stages of the disease, when therapeutic efforts are most efficacious.

The microdystrophin gene transfer therapy from Solid Biosciences has shown protein expression in the first 3 patients dosed. As a result, the company is expediting the planned dose-escalating activities.

The professor of pediatrics at Northeast Ohio Medical University and the director of the NeuroDevelopmental Science Center at Akron Children’s Hospital provided insight into the therapeutic landscape of PMM.

A phase 3 trial of the therapy is ongoing after a number of positive signs from earlier assessments. Thus far, the tetrapeptide has shown potential to become the first treatment for PMM.

Neurology News Network for the week of February 2, 2019.

Approximately 60% of the patients with ALS are already excluded from clinical trial participation at the day of diagnosis, but investigators think that 60% is an underestimate.

CAP-1002 is the only therapy to show reduction in myocardium scar and an improvement of localized cardiac function in late-stage Duchenne accompanied cardiomyopathy.

Neurology News Network for the week of January 26, 2019.

The novel myeloperoxidase inhibitor was previously licensed by AstraZeneca, but its development has since been taken over by Biohaven, which received IND acceptance from the FDA.

The regulatory agency has informed Cytokinetics that the use of the 6-minute walk test will be an acceptable efficacy outcome measure for the skeletal muscle troponin activator.

The agency is anticipating upward of 200 INDs per year by 2020 and between 10 and 20 cell and gene therapy approvals per year by 2025.

A small trial has suggested that patients with myasthenia gravis and dysphagia are not properly assessed by the common clinical scales for these swallowing problems and their risk of silent aspiration.