The Global Crisis of Misdiagnosis and Challenges in Care for Rare Diseases: Sumaira Ahmed
January 30th 2024The founder and executive director of the Sumaira Foundation discussed the increasing amount of misdiagnosis in rare diseases such as NMOSD and how it impacts these patients in terms of care and support. [WATCH TIME: 2 minutes]
Positive Phase 2 Data Announced for MOMENTUM Study of Duchenne Agent SRP-5051
January 30th 2024Over a 28-week treatment period, the investigational agent demonstrated significantly greater increase in dystrophin expression and exon skipping than previously approved eteplirsen, Sarepta’s first therapy for Duchenne muscular dystrophy.
Insights on GBA1-Targeting Agent GT-02287 for Parkinson Disease: Joanne Taylor, PhD
January 29th 2024The senior vice president of research at Gain Therapeutics talked about preclinical findings from a study assessing GT-02287, an agent in development for the treatment of GBA1 Parkinson disease, and future plans for clinical trials. [WATCH TIME: 7 minutes]
Patient Perspective on the Health Canada Approval of Inebilizumab for NMOSD: Sumaira Ahmed
January 29th 2024The founder and executive director of the Sumaira Foundation shared her patient perspective of Health Canada’s approval of inebilizumab in terms of its implications for patients and how it paves the way for future treatments in NMOSD. [WATCH TIME: 3 minutes]
Easing Neurosurgery With Newly Approved SmartFrame OR Stereotactic System: Joe Burnett
January 28th 2024The chief executive officer of ClearPoint Neuro discussed how the company’s SmartFrame OR Stereotactic System can facilitate neurosurgery and the next steps in integrating it into clinical settings. [WATCH TIME: 4 minutes]
Low-Dose Benzodiazepines Linked With Worsened Disease Progression in Progressive Supranuclear Palsy
January 27th 2024Even after adjusting for indications for benzodiazepine administration and baseline disease severity, results showed greater patient disease worsening while on such medications, regardless of duration and dose.
Caregiving Youth: A Call to Action for Improved Support
January 27th 2024A systematic approach to understanding the needs of the caregiving youth—a growing and less-studied population of caregivers—is needed to better tailor interventions, provide support, and improve care to patients and their families.
Health Canada Approves Inebilizumab as Treatment for Neuromyelitis Optica Spectrum Disorder
January 26th 2024In recent news, Health Canada approved inebilizumab for adult patients with neuromyelitis optica spectrum disorder who are anti-aquaporin-4 antibody positive, using results from the N-MOmentum trial as the basis for the approval.
Neuroprotectant Mechanism of Action of Stroke Exosome AB126: Stephen From
January 26th 2024The chief executive officer of Aruna Bio discussed the mechanism of action behind AB126 and how its anti-inflammatory and neuroprotective properties may be effective in treating acute ischemic stroke. [WATCH TIME: 4 minutes]
Long-term Tocilizumab Therapy Effective in Regulating Lymphocytes and Neutrophils in NMOSD
January 25th 2024A recent analysis revealed a reduction in regulatory lymphocyte subsets in patients with NMOSD before tocilizumab therapy, with subsequent restoration to normal levels after 1 year treatment.
Case Study Shows ITPR1 Variant Associated With Spinocerebellar Ataxia Type 29
January 25th 2024In a recent case study, clinical exome sequencing exposed a heterozygous missense mutation in the ITPR1 gene where patients showed craniofacial abnormalities as well as spinocerebellar ataxia-like syndrome.
Insights on Seizure Therapy and Future Plans in Epilepsy Research: Iryna Leunikava, MSc
January 25th 2024The European evidence generation strategy lead for epilepsy and rare syndromes at UCB described some details about further epilepsy research and other explorations in the next few years. [WATCH TIME: 3 minutes]
Enhancing Methodologies for Comprehensive Patient Outcome Analysis in Epilepsy: Derek Ems, MPH, CPHQ
January 25th 2024The health economics outcomes research specialist at UCB discussed the need for improved methodologies to have a more accurate analysis of patient outcomes beyond what administrative databases can provide. [WATCH TIME: 2 minutes]