Talk to Our
Neuromuscular Experts



The sense of urgency to free patients from the punishing burden of rare neuromuscular diseases has never been greater as the pursuit of genetic therapies presents a bold new hope. To realize the potential of these therapies, many complex clinical development challenges must be overcome.

The Right Experience in Rare Neuromuscular Trials and Innovative Therapies to Advance Your Studies

Our Experienced Neuromuscular Teams Are Ready

PPD has the medical, regulatory and operational knowledge to help safely speed your treatments to patients with neuromuscular diseases. Our neuromuscular team has the global expertise required to help you overcome patient, site and regulatory challenges, gained from experience serving both emerging biotechs and larger pharma.

“My DMD patients are so optimistic about potential therapies, and with the acceleration of decentralized trial technology and the further adoption of home health, so am I”

Guiding Your Innovative Cell and Gene Therapies  

Gene therapy is bringing new hope for patients with neuromuscular disorders. However, the many unknowns in gene therapy further complicate the average challenges of drug development. Our guidance begins at the pre-clinical stage to maximize resources throughout the development process.

PPD has the end-to-end experience and capabilities in cell and gene therapy to help you better estimate how regulators, payors and patients will react to your therapy.

Clinical execution Planning and operationalize studies with unique challenges – e.g. training and monitoring sites for complex medicinal product administration, oversight of IP logistics and cross-border recruitment.
Payor strategy We will work with you to build your value case and identify innovative payment methods, to persuade payors to grant patients access to your therapy.
Regulatory approval A thoughtful evidence strategy from early development is critical, as many gene therapy studies are single-arm and most will require natural history studies and registries, as well as long-term follow-up of all dosed patients for up to 15 years.

Early Engagement Drives Success in Neuromuscular Trials

Trials for neuromuscular disorders such as Duchenne muscular dystrophy (DMD) and amyotrophic lateral sclerosis (ALS) face significant complexities: geographically dispersed patients, few sites with the requisite experience, diagnostic heterogeneity, and widely varying in-country care and health authority regulations.

With strong neuromuscular and rare disease experience, PPD’s operational, medical and regulatory experts are ready from the earliest stages of development to guide your study design to ensure you meet the stringent ethics requirements, while achieving enrollment targets, assessing the right endpoints and managing costs.

Increased focus on the cost of rare disease treatments

New rare disease products are improving outcomes for patients, but health system budgets haven’t caught up with the high prices of these therapies. Payors are managing the cost by restricting eligibility and reimbursement only to patients who are likely to have a significant benefit over standard of care. PPD has the expertise to help you address questions like these with confidence:

  • What are the characteristics and disease outcomes of target patient populations?
  • What value assessments are likely to drive the greatest impact for regulators and payors?
  • How can we plan for and implement the right study design?
  • How can we maximize access for patients?

Together in partnership, we will navigate the complex neuromuscular development journey with a shared goal in mind: to bring independence and freedom to patients with neuromuscular disorders.   

Speak with one of our specialists to learn how we can help accelerate your therapy and free patients from neuromuscular disorders.