uniQure Receives DSMB Recommendation to Resume Higher Dosing of AMT-130 in Huntington Trial
November 13th 2022The 26-patient trial includes 10 individuals with early-manifest Huntington disease on low-dose AMT-130 and 16 in the high-dose cohort, which will continue enrollment after a DSMB found no further safety concerns.
The Sumaira Foundation Providing Access to Care for Patients with NMOSD: Sumaira Ahmed
November 13th 2022The founder and executive director of the Sumaira Foundation discussed her experience living with NMOSD and the global organization she created to help patients with the same condition. [WATCH TIME: 6 minutes]
Critical Takeaways, Learning Experiences From the Tofersen Trials in ALS: Timothy Miller, MD, PhD
November 11th 2022The codirector of the ALS Center at Washington University School of Medicine provided perspective on the lessons learned from the trials of tofersen, as it aims to become the first FDA-approved therapy specific to SOD1 ALS. [WATCH TIME: 4 minutes]
Progress for Patients With Pediatric MS, Fingolimod's Risk-Benefit Profile: Brenda Banwell, MD
November 11th 2022The chief of neurology and codirector of the neuroscience Center at the Children's Hospital of Philadelphia spoke about her presentation at ECTRIMS 2022 on the treatment of pediatric MS. [WATCH TIME: 5 minutes]
Role of Tofersen Among Other Previously Approved ALS Therapies: Timothy Miller, MD, PhD
November 10th 2022The codirector of the ALS Center at Washington University School of Medicine in St. Louis provided background on how tofersen would be used alongside other ALS medications if approved. [WATCH TIME: 3 minutes]
Use of Nanoparticles to Combat Neurodegenerative Disease
November 10th 2022Available neurodegenerative disease treatments are generally unsafe and ineffective at penetrating the blood-brain barrier, though the use of nanoparticles can provide improved penetration and exert a neuroprotective effect.
Institutional Perspectives in Neurology, Chaired by Robert T. Naismith, MD
November 10th 2022Chaired by Robert T. Naismith, MD, of Washington University in St. Louis, the presentations also feature Salim Chahin, MD, MSCE; Anne H. Cross, MD; Gregory F. Wu, MD, PhD; and Matthew R. Brier, MD, PhD. [WATCH TIME: 1 hour, 55 minutes]
NurOwn Stromal Cell Therapy Continues to Show Positive Data in Less Severe ALS
November 9th 2022After removing participants at higher risk of reaching a floor effect of the ALSFRS-R, those treated with NurOwn demonstrated a higher rate of clinical response and less function lost across 28 weeks than placebo.
Advocacy and Empowerment: Finding Your Advocacy Community
November 9th 2022The Palatucci Advocacy Leadership Forum, or PALF, sponsored by the American Academy of Neurology, gives neurologists and trainees tools to successfully advocate for their ideas and develop their identity as physician advocates.
Understanding Tofersen’s Impact on SOD1 ALS, Neurofilament Light: Timothy Miller, MD, PhD
November 9th 2022The codirector of the ALS Center at Washington University School of Medicine in St. Louis discussed the long-term data of VALOR and its open-label extension assessing tofersen (Biogen) in SOD1 ALS. [WATCH TIME: 4 minutes]