Neuromuscular

The treatment was efficacious in patients with AQP4-IgG-positive NMOSD, which accounts for nearly three-quarters of the population with the disorder, for which there is currently no approved treatment.

The phase 2 STARS trial was the first time in 50 years that an overall improvement in patients with Angelman syndrome was observed, in this instance with a once-daily dose of gaboxadol, also known as OV101.

In a pair of phase 2 trials and a phase 1 study, patients with SMA types 1 and 2 treated with the gene therapy displayed a number of motor milestone achievements and a prolonged event-free survival rate.

Interim results of the phase 1/2 study show statistically significant reductions in SOD1 protein levels in cerebrospinal fluid, suggesting lower concentrations in the brain and spinal cord, as well.

Changes in MS diagnostic criteria and the availability of highly effective DMTs has deemed the risk for postpartum relapse nearly irrelevant for the majority of patients with MS.

Although highly efficacious DMTs have improved outcomes for a growing number of patients, the majority of people with MS are still subjected to the day-to-day consequences of the disease’s symptoms, which often have a more debilitating effect on the patient than the disease itself.

The investigational candidate is the subject of the 2-part phase 2/3 SUNFISH trial in young adult and pediatric patients with type 2 and type 3 SMA.

Over the course of approximately 3 years, Hammersmith Functional Motor Scale-Expanded and Upper Limb Module scores, as well as 6-Minute Walk Test distances, improved significantly in patients with SMA type 2 and type 3.

Infants with SMA—who have elevated pNF-H levels—experienced significant drops in pNF-H levels when treated with nusinersen (Spinraza, Biogen), suggestive of pNF-H’s potential as a biomarker of disease activity and treatment response.

The approval of nusinersen in 2016 to treat spinal muscular atrophy marked an important milestone in efforts to treat the disease, but even more breakthroughs could be coming soon.

Responses from 1300 physicians suggest that increasing patient load and adding ancillary services may be the key to increasing annual income.

Neurology News Network for the week of April 20, 2019.

With 2 targeted treatments on the market, therapeutic development may now focus on early detection.

The phase 1 results support the initiation of the phase 2/3 clinical trial which is expected to initiate in July 2019.