Neuromuscular Disease Drug Development
Accelerate your neuromuscular disease study with Medpace’s noted medical and regulatory experts, highly experienced clinical trial management teams, central labs, and imaging core labs
Diseases characterized by neuromuscular degeneration are particularly challenging when it comes to study design and endpoint selection. That’s why you need a trusted partner that has the scientific leadership, operational execution and comprehensive services in place to help accelerate your compound to approval. Our physicians and staff have experience in rare disease and in particular diseases that impact neuromuscular function such as:
- Duchenne Muscular Dystrophy
- Adult forms of Muscular Dystrophy
- Pompe’s disease
- Multiple Sclerosis
- Charcot Marie Tooth Disease
- Spinal Muscular Atrophy
The Medpace Difference for Neuromuscular Disease Studies
- In-house neurologists, pediatricians, regulatory experts, and clinical operations professionals that are skilled in neuromuscular disease studies
- Specialized project teams that can utilize novel endpoints to ensure efficient study execution from study start up through completion of the clinical study report
- Extensive expertise in gene editing, gene transfer technology and other advanced therapies along with the additional submissions required to ensure regulatory compliance
- Wholly-owned imaging core lab with an end-to-end suite of global imaging services ensuring imaging components are seamlessly integrated into the complex structure of the overall trial
- Global central lab with safety and biomarker analysis to support neuromuscular disease studies
- Flexible, highly-customizable ePRO solution that is fully-integrated into ClinTrak®, Medpace’s proprietary data management system
- Multi-dimensional recruitment model that enables us to implement customized recruitment and retention strategies that identify, recruit, and retain challenging patient populations
Central Laboratory Capabilities
With laboratories in the US, Europe, China and Singapore, Medpace Labs has the global reach and capabilities to conduct neuromuscular disease studies in concert with our full-service CRO or as a standalone service. Key highlights include:
- Extensive menu of biomarkers that use state of the art techniques, and provide results that enable patient stratification and improve prediction of drug efficacy and safety.
- Scientific engagement throughout the development lifecycle from our global team of experts who have extensive central laboratory operations experience
- Over 19,000 square feet of biorepository services located at our four global central laboratory locations
- In-house logistical team that manages all shipments of collection kit supplies and laboratory manuals to clinical sites
- Seamless integration with a robust network of laboratory partners, some which specialize in CNS testing such as CSF biomarkers and genotyping.
Core Imaging Expertise for Neuromuscular Disease Studies
High-quality image acquisition and interpretation is crucial for the success of trials relying on neuroimaging for patient selection or as a primary endpoint. Medpace provides centralized neuroimaging expertise, led by radiologists and neurologists with decades of clinical and trial experience.
Medpace Imaging Core Lab provides an end-to-end suite of imaging services to enhance and expedite biopharmaceutical and imaging contrast agent development, including a broad spectrum of imaging biomarkers for DMD trials. Capabilities include:
- MR Spectroscopy
- T2 Mapping
- Muscle Fat Fraction
- PDFF Complex
- MR Elastography
