Gene Therapy Fast Tracked for APOE4 Associated Alzheimer Disease
The investigational adeno-associated virus mediated gene therapy is being developed to deliver the protective APOE2 gene to the CNS.
Neurologic Disorders
Nulibry Now Available for Molybdenum Cofactor Deficiency Type A
MoCD Type A is a rare, inborn error of metabolism caused by mutations in the MOCS1 gene that results in toxic sulfite accumulation in the brain.
AAN: Race May Affect Response to Anti-CD20 Therapy for MS, NMOSD
B-cell repopulation occurs more often in African American vs White patients between 6 and 12 months after anti-CD20 infusion
Ponvory Now Available for Relapsing Forms of Multiple Sclerosis
Ponvory is a once-daily oral selective sphingosine-1-phosphate receptor 1 modulator.
Pamrevlumab Fast Tracked for Duchenne Muscular Dystrophy
Pamrevlumab is a first-in-class fully human antibody that works by inhibiting the activity of connective tissue growth factor.
Xywav Gets Priority Review for Idiopathic Hypersomnia
The sNDA is supported by data from a phase 3 study that assessed Xywav in adults with idiopathic hypersomnia.
Considerable Neurologic, Psychiatric Morbidity Reported After COVID-19
Estimated incidence of neurological or psychiatric diagnosis was 33.62 percent among 236,379 patients
FDA Rejects Pimavanserin sNDA for Dementia-Related Psychosis
The sNDA submission was supported by data from the pivotal phase 3 HARMONY study.
FDA: Risk of Arrhythmias With Lamotrigine in Patients With Heart Disease
Lamotrigine is indicated as monotherapy or adjunctive therapy for epilepsy and for use as maintenance treatment in patients with bipolar I disorder.
Tongue-Delivered Neuromodulation Therapy Gets FDA Clearance for MS-Related Gait Deficit
PoNS is a portable, noninvasive, prescription-only device intended to be used as an adjunct to a supervised therapeutic exercise program in patients 22 years of age and older.
