Genetic Targets Invigorate Research for Duchenne Muscular Dystrophy
February 22nd 2019Over 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 Success of Risdiplam and Importance of Screening Infants for SMA
February 22nd 2019The 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.
Antiepileptic Drugs Not Linked to an Increased Risk of Dementia
February 21st 2019AED use was not significantly associated with dementia risk in patients followed by general practitioners and neuropsychiatrists, however, the potential harmful effects of levetiracetam generic brands on cognition should be further investigated.
Reldesemtiv Displays Promise Over Predecessor for ALS, Currently in Phase 2b Trial
February 20th 2019The 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.
FDA Issues Warning About Misuse of Young Donor Plasma Infusions
February 19th 2019A joint statement from the FDA commissioner and the director of the agency’s Center for Biologics Evaluation and Research noted the product is being offered at a variety of establishments as a treatment for conditions for which its benefits are unproven.
Advances Answer Some Questions in Excessive Sleepiness, But Raise New Ones
February 19th 2019The 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.
MPRI 2.0 Accurately Differentiates Parkinson Disease and Progressive Supranuclear Palsy
February 19th 2019A new investigation has suggested that the Magnetic Resonance Parkinsonism Index 2.0 can accurately predict the clinical evolution toward a progressive supranuclear palsy-parkinsonism phenotype, differentiating it from Parkinson disease.