Victoria Johnson

Caution is warranted for older patients with Duchenne muscular dystrophy who are receiving gene therapy, according to details of a patient death in a single patient trial.

The FDA lifted its clinical hold on Astellas Pharma's FORTIS trial for evaluating AT845 in adults with late-onset Pompe disease.

The AAV1 gene therapy, PBFT02, is being assessed in a phase 1/2 trial called upliFT-D (NCT04747431), which is planned to take place over the course of 24 months in patients with frontotemporal dementia with granulin mutations.

The deaths, occurring shortly after treatment infusion, were because of acute liver injury, which is a known risk associated with the Novartis gene therapy.

Children with AADC receiving treatment developed motor function and cognitive skills that persisted through up to 10 years of follow-up.

Patients with Duchenne muscular dystrophy receiving the treatment had a decline in Mid-level Performance of Upper Limb scores in the phase 2 trial of the cell therapy.

In addition to the uplifted phase 1 study, Novartis also initiated the phase 3 STEER study, which will evaluate OAV-101 in treatment-naive patients with SMA type 2 aged between 2 and 18 years old.

While the relationship between sleep and cognition needs to be further studied, sleep-based interventions may be a future approach to help prevent cognitive decline.

Santhera and ReveraGen announced findings of the phase 2b VISION DMD study of the investigational therapy.

Bayer provided an update on their phase 1 and 1b studies of dopaminergic neurons and GDNF gene therapy, respectively, for the treatment of PD.

Results from the START study of medication switching in narcolepsy were presented at the 2021 SLEEP Annual Meeting.

Data presented at SLEEP 2021 also showed the treatment was associated with significant weight loss and reduction in BMI compared to placebo.

A post-hoc analysis of the phase 2/3 trial of rimegepant revealed early and sustained efficacy compared to placebo, with significant reductions in weekly migraine days.

Investigators conducted a 9-month study that demonstrated the correlation of the MBI, calculated as the sum of headache days times the maximum intensity of headache on each headache day, with satisfaction of migraine status.

Recent data presented at the AHS annual meeting showed clinically meaningful improvements in MMD and MHD in patients with both chronic and episodic migraine.

A post-hoc analysis of 4 clinical trials of galcanezumab found no differences between dose groups of galcanezumab or placebo in rates of ‘wearing off’ effect.

The reported reasons included wanting to take care of symptoms themselves, concern that migraine will not be taken seriously, and believing migraines are not severe or painful enough to seek care.

Investigators’ analysis of a subset of patients with insufficient response from the CENTURION study suggests that the acute agent from Eli Lilly offers potential in this population.

Experts were in agreement that the criteria should include PTH phenotype, TBI mechanism and TBI comorbidities.

The nasal delivery of dihydroergotamine also showed a lower incidence of nausea than IV-administrated dihydroergotamine.

Rimegepant (Nurtec ODT; Biohaven) had the lowest number needed to treat for sustained pain freedom from migraine.

AC Immune SA also continues to develop an improved formulation of the investigational agent.

Robert Zivadinov, MD, PhD, professor of neurology and director of the Buffalo Neuroimaging Analysis Center, discussed the post-hoc analysis of the ORATORIO trial he and colleagues conducted.

Through surveys and an international meeting, the International Society for CNS Clinical Trials Methodology developed consensus criteria for NCD-specific apathy.

Richard Gershon, PhD, vice chair for research at the Northwestern University Feinberg School of Medicine, discussed the development of the mobile toolbox battery to track cognitive decline.

The American Heart Association/American Stroke Association has published updated guidelines for the prevention of secondary stroke.

The long-term follow up of the phase 1 START trial found that previously achieved motor milestones were preserved over a 5-year period.

Stephen Rao, PhD, ABPP-Cn, the Ralph and Luci Schey Chair and Director of the Schey Center for Cognitive Neuroimaging at Cleveland Clinic, discussed the development of the MSPT.

A 5-year, post-approval study of the remedē System confirmed results of the pivotal trial.

Researchers could not identify any risk factors for contracting COVID-19 in the population with epilepsy.