Child in wheelchair with nurse

Neuromuscular Disorder Studies

Bringing hope and freedom to patients with neuromuscular disorders

Gene therapies offer a new era of hope for patients suffering from rare neuromuscular diseases, and the PPD™ clinical research business of Thermo Fisher Scientific is prepared to connect patients with these often-debilitating disorders to clinical trials for potentially life-changing treatments. Creating these therapies presents many complex developmental, clinical and regulatory challenges, which are best managed with the support of an experienced team of experts.

As your partner, we enable you to navigate and overcome these challenges with access to patient-centric care and services that accelerate study recruitment and increase participant retention. Learn more about our extensive patient care offerings, including proactive, tailored concierge services that reduce the individual burden for neuromuscular disease patients and their caregivers.

Experience in neuromuscular trials and innovative therapies to advance your studies

40+ neuromuscular studies from early first-in-human studies to post-approval, as well as registry and natural history studies
Includes indications such as DMD (ambulatory & non-ambulatory), myasthenia gravis, FSHD, CIDP, MELAS, SMA
130+ cell and gene therapy studies that include lentivirus, various AAVs, gene editing and naked DNA delivery
Includes neuromuscular indications
Experience with infusion directly into the central nervous system

Experienced teams ready to guide your neuromuscular disorder clinical trails

To safely increase the speed with which your treatments are delivered to patients with acquired or genetic conditions that affect some part of the neuromuscular system — such as spinal muscular atrophies — you need a partner with deep medical, regulatory and operational knowledge in neuromuscular disorders. We have extensive experience in treating rare pediatric conditions at our Rare Disease and Pediatric Center of Excellence (COE).

With the global expertise and robust, indication-specific site partnership network required to help you overcome site and regulatory challenges, our neuromuscular team can leverage the expertise gained from serving both emerging biotechs and multinational pharma companies using a strategy that puts the patient first.

Doctor speaking with child and her mother in a clinical setting.

Patient services that drive trial success and positive health outcomes

Because patients with rare neuromuscular disorders often face symptoms like muscle weakness and muscular atrophy that can make it challenging to move around, it’s of particular importance that patients in neuromuscular trials have access to flexible remote and hybrid study opportunities and logistical support for travel.

Make it easier for those affected by neuromuscular diseases to handle the intricacies of taking part in clinical trials with our robust patient outreach services. We assign a concierge to each patient at an early stage so that they can receive consistent and proactive support, which is designed to reduce individual burden and increase patient retention as they participate in your neuromuscular clinical trial.

These tailored patient and caregiver engagement services include:

  • Full-service booking and coordination of patient transportation to and from study sites that provide convenience for the patients, access to reliable transport, and travel expense verification
  • Flexible options for patient reimbursement in more than 150 currencies
  • Home health care options including partnering with remote staff
  • Connection to flexible protocols for digital and decentralized trials and hybrid approaches using mobile pages, wearables, telehealth and e-consent

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Guiding leaders in innovative cell and gene therapies for neuromuscular diseases

The great potential of novel cell and gene therapies to treat neuromuscular disorders also comes with a number of unknowns that complicate and potentially compound the existing challenges of drug development. Our guidance begins at the preclinical stage in order to maximize resources during the entire drug development process.

You can better understand and estimate how regulators, payers and patients will react to your treatment with our end-to-end support and capabilities in cell and gene therapies.

Clinical execution Plan and operationalize studies with unique challenges, including training and monitoring sites for complex medicinal product administration, overseeing IP logistics and performing cross-border recruitment.
Payer strategy Build your value case and identify innovative payment methods to make payments more accessible to patients and encourage payers to grant access to your therapy.
Regulatory approval Establish a thoughtful, evidence-based strategy from early development of your therapeutic as a critical part of building out the often-required natural history studies and registries through long-term follow-up studies of dosed patients for up to 15 years.

Accelerate success with early engagement for your neuromuscular disorder trials

Trials for neuromuscular disorders such as Duchenne muscular dystrophy (DMD) and amyotrophic lateral sclerosis (ALS) face significant complexities, including:

  • Diagnostic heterogeneity
  • Geographically dispersed patients
  • Limited sites with the requisite indication-specific experience
  • Widely varying in-country care and health authority regulations

With strong neuromuscular and rare disease expertise, our operational, medical and regulatory teams are ready to guide your study design from the earliest stages of development. The guidance ensures that you meet the stringent ethics requirements while achieving enrollment targets, assessing the right endpoints and managing costs.

Increased focus on cost for 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. Payers are managing the cost by restricting eligibility and reimbursement to patients who are likely to have a significant benefit in their standard of care.

To ensure that patients at all socioeconomic levels can get access to your treatments for rare neuromuscular disorders, we can help you address questions like these with confidence:

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

Reach out about your neuromuscular clinical trial

As your partner, we will navigate the complex neuromuscular development journey together with a shared goal in mind: to bring independence to patients with rare neuromuscular disorders. Speak with one of our specialists today to learn how we can accelerate your therapy and treat those who suffer from muscle disorders from the onset.