February 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.

More recently, investigators have discovered that catechol-O-methyltransferase inhibitors can prolong the effects of levodopa, thereby limiting the off-time phenomenon.

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 field of neurology is gaining a more robust understanding of some of the mechanisms at play in excessive daytime sleepiness, but the progress has not spread evenly across different sleep disorders.

As many patients with migraine and other disorders are resistant to currently available options, this increase in device development has brought forward additional path­ways to treatment for patients.

The new funding initiatives and innovative programs to expedite research efforts could hasten the search for biomarkers of Alzheimer disease in the blood and, ultimately, open the door to easier, quicker, and more affordable diagnostic methods.