Silas Inman

Over a 2-year period, SRP-9003 elicited sustained protein expression in muscle tissue and stabilized North Star Assessment for Dysferlinopathies scores.

No differences were observed in the magnitude of ubrogepant treatment effect between defined triptan subgroups.

Atogepant significantly reduced mean monthly migraine days compared to placebo across a number of doses and was generally well-tolerated, with no treatment-related serious adverse events.

A 75 mg orally dissolving tablet formulation of the calcitonin gene-related peptide receptor antagonist met both co-primary end points of freedom from the patient-indicated most bothersome symptom and freedom from pain at 2 hours.

ITI-214 showed favorable safety with early signs of improvement in motor symptoms for patients with Parkinson disease

The CGRP inhibitors are poised to transform the treatment paradigm for patients with migraine and cluster headache.

VY-AADC01 demonstrated promising early signs of activity and safety for patients with Parkinson disease.

Several novel therapies in early stage development are beginning to show promise for patients with Huntington disease.

A novel mesenchymal stem cell therapy produced intriguing phase II data and has moved into a randomized, pivotal phase III clinical trial.

Cellular therapies have the potential to make a meaningful difference for patients with neurological disorders, if used correctly.

IncobotulinumtoxinA significantly reduced unstimulated salivary flow rate for patients with sialorrhea.

Patisiran maintained improvements in mNIS+7 and rapidly halted neurologic disease progression in hATTR amyloidosis.

Subcutaneous immunoglobulin is an effective and well-tolerated alternative to intravenous immunoglobulin for myasthenia gravis.

Eculizumab showed sustained activity and tolerability through 3 years for generalized myasthenia gravis.

Novel biomarkers are being explored to help expedite the development of new treatments for Alzheimer disease.

Allogeneic mesenchymal stem cells manufactured by Longeveron are advancing through phase I and II clinical trials.

The mesenchymal stem cell therapy passed an interim safety analysis for the first 31 patients with amyotrophic lateral sclerosis.

Several clinical trials are examining the role of stem cells, inflammation, and other pathways in ALS.

A third-party manufacturing issue has delayed a phase I/IIa study for Sarepta Therapeutics.

The addition of ZX008 to background stiripentol significantly reduced the number of convulsive seizures experienced by children and young adults with Dravet syndrome.

The FDA has accepted a supplemental new drug application for sodium oxybate as a treatment for cataplexy and excessive daytime sleepiness in children with narcolepsy.

The autologous stem cell therapy is currently in a phase III trial.

Rimegepant significantly reduced pain and the most bothersome symptom while showing an "excellent" safety profile for patients with acute migraine attacks.

The FDA had accepted a new drug application for APL-130277, a sublingual film version of apomorphine, as a treatment for OFF episodes in patients with Parkinson disease.

The FDA has granted a priority review designation to perampanel for 2 potential new indications for pediatric patients with seizures.

The first-in-class anti-CGRP monoclonal antibody erenumab has gained FDA approval for the prevention of migraines.

The FDA has expanded the indication for fingolimod to include the treatment of children and adolescents age 10 years and older with relapsing multiple sclerosis.

The treatment is the first patient-infused subcutaneous formulation of immunoglobulin to gain approval for CIDP.

The FDA has accepted an application for solriamfetol as a treatment for excessive sleepiness associated with narcolepsy or obstructive sleep apnea, with a PDUFA action date set for December 20, 2018.

The FDA had accepted a new drug application for the inhaled levodopa therapy for the treatment of OFF period symptoms in patients with Parkinson disease who are taking a carbidopa/levodopa regimen.