A5037213636- Neurogenetic and Muscular Disorders Research
- Cardiomyopathy and Myosin Studies
- Muscle Physiology and Disorders
- Genetics and Neurodevelopmental Disorders
- Viral Infections and Immunology Research
- RNA Interference and Gene Delivery
- Cancer, Lipids, and Metabolism
- Nuclear Structure and Function
- Radiopharmaceutical Chemistry and Applications
Boston Children's Hospital
2024
Johns Hopkins University
2024
Harvard University
2024
Stanford University
2024
The University of Texas Southwestern Medical Center
2024
American Physical Therapy Association
2024
Scholar Rock (United States)
2021-2024
Pfizer (United States)
2024
Karyopharm Therapeutics (United States)
2018
Currently approved therapies for spinal muscular atrophy (SMA) reverse the degenerative course, leading to better functional outcome, but they do not address impairment arising from preexisting neurodegeneration. Apitegromab, an investigational, fully human monoclonal antibody, inhibits activation of myostatin (a negative regulator skeletal muscle growth), thereby preserving mass. The phase 2 TOPAZ trial assessed safety and efficacy apitegromab in individuals with later-onset type 3 SMA.
11512 Background: Locally advanced DDLS is incurable with an overall survival of 11 – 20 mo palliative therapies. Ideal imaging criteria for efficacy currently undefined. Selinexor (S) oral, selective inhibitor nuclear export that specifically blocks exportin 1, leading to the accumulation and reactivation tumor suppressor proteins. S demonstrated anti-tumor activity against in preclinical studies a Ph 1b study patients (pts) soft tissue sarcomas. Methods: Eligible pts had progressive...
<h3>Objective:</h3> To evaluate patient treatment effects beyond TOPAZ trial endpoint measures to provide a deeper understanding of the potential benefit apitegromab. <h3>Background:</h3> SMA is caused by deletion or mutation in SMN1 gene and characterized motor neuron deterioration that contributes progressive muscle weakness loss function. Current SMN treatments have shown improvements function; however, limitations remain contribute unmet needs. In Phase 2 (TOPAZ, NCT03921528), we...