Publications by authors named "M Freimer"
Article Synopsis
- The study aimed to assess the safety and effectiveness of caprylate/chromatography-purified intravenous immunoglobulin (IGIV-C) in patients with generalized myasthenia gravis (MG) compared to a placebo.
- A total of 62 patients participated in a randomized trial, where IGIV-C showed a numerical improvement in certain outcomes like activities of daily living, although most primary and secondary outcomes did not reach statistical significance.
- While IGIV-C was generally well-tolerated, the small size of the study suggests the need for further research to better understand its potential as a maintenance therapy for MG.
Article Synopsis
- Generalized myasthenia gravis (gMG) is a chronic disease that significantly affects patients' quality of life and highlights the need for better treatments.
- The ongoing phase III study evaluates the long-term safety and effectiveness of the drug zilucoplan in patients with acetylcholine receptor autoantibody-positive gMG.
- Results from 200 patients showed that a significant majority experienced treatment-emergent adverse events, but those receiving zilucoplan showed continued improvements in daily living activities over 60 weeks, especially after switching from a placebo.
Background: Facioscapulohumeral muscular dystrophy is a hereditary progressive myopathy caused by aberrant expression of the transcription factor DUX4 in skeletal muscle. No approved disease-modifying treatments are available for this disorder. We aimed to assess the safety and efficacy of losmapimod (a small molecule that inhibits p38α MAPK, a regulator of DUX4 expression, and p38β MAPK) for the treatment of facioscapulohumeral muscular dystrophy.
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- Fatigue significantly affects patients with myasthenia gravis (MG), and its impact can be measured using the Neuro-QoL Short Form Fatigue scale, which converts scores into T-scores for better clinical understanding.
- In the Phase 3 RAISE study, patients with MG were treated with zilucoplan or a placebo, with those completing the study offered ongoing treatment in RAISE-XT.
- Results showed that zilucoplan led to significant improvements in fatigue scores and severity, with benefits lasting up to 60 weeks after treatment started.
Background: Patients with neurologic diseases have complex medical needs and may benefit from the addition of clinical pharmacists in their care.
Objectives: This study aimed to describe integration and benefit of clinical pharmacists in neuroimmunology and neuromuscular clinics at an academic medical center.
Methods: This retrospective chart review evaluated patients initiated on a neurology medication for a neuroimmunology or neuromuscular disease state before and after pharmacist integration in neurology clinics.