Itvisma should only be administered intrathecally using a lumbar puncture by health care professionals experienced in performing the procedure. The Food and Drug Administration has approved Itvisma ® ...

Credit: Novartis. The approval was supported by data from phase 3 STEER study, which enrolled patients with SMA who were treatment-naive and able to sit but never able to walk independently. Itvisma ...

Take advantage of TipRanks Premium at 50% off! Unlock powerful investing tools, advanced data, and expert analyst insights to help you invest with confidence. Study Overview: Alnylam Pharmaceuticals, ...

THE neurologic manifestations of Central-nervous-system involvement in acute leukemia have been very well described. 1–7 Although involvement of the brain is not rare 7,8 greater emphasis has been ...

Department of Molecular and System Pharmacology, Graduate School of Pharmaceutical Sciences, Kyushu University, Fukuoka, Japan Division of Molecular Neuroimmunology, Medical Institute of Bioregulation ...

Multiple sclerosis (MS) is a heterogeneous disease, in both clinical presentation and neuro-immunopathology. Proteins secreted into the cerebrospinal fluid (CSF) can serve as molecular proxies of ...

Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) being an oral daily medication and nusinersen (Spinraza) being administered ...

Particulate contamination has sparked the recalls of numerous injectable drugs over the years, with Amneal’s hospital med ropivacaine now the latest to fall victim to the trend. Amneal is recalling ...

FOLLOWING the demonstration that penicillin exhibits a remarkable antibacterial effect against organisms frequently causing meningitis, there immediately arose the question of the most effective mode ...

After bringing Zolgensma to market in 2019 as the first gene therapy for spinal muscular atrophy, Novartis is back with an intrathecal formulation intended for older patients. Six years ago, the FDA ...

OAV101 IT, a version of the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) that’s administered into the spinal canal, can be safely given to people with spinal muscular atrophy (SMA) who were ...