Advancing Neuromuscular Research
Deep Expertise in Neuromuscular and Neurodegenerative Trials
Ergomed brings proven global leadership to neuromuscular clinical trial design and execution—from first-in-human studies to Phase 3 trials and post-marketing safety programs. Our expertise in DMD, SMA, and ALS research is backed by our operational excellence, trusted investigator relationships, and a track record of success.
Advancing Neuromuscular Research with Precision, Partnership and Patient Focus
Ergomed empowers biopharma sponsors to bring rare disease therapies, including those for neuromuscular and neurodegenerative disorders, to market faster, smarter, and with greater certainty.
Post-Market Safety & Risk Management in DMD
Our latest case study highlights how Ergomed Consulting supported a pharmaceutical company specializing in rare neurological diseases to manage long-term safety and sustain market performance for a therapy treating Duchenne muscular dystrophy (DMD).
Engaging Patients and Advocacy Communities with Purpose
We foster trust across the care ecosystem by involving patients, their care partners, and patient advocacy organizations throughout the trial lifecycle. Our team includes rare disease advocates and trial-experienced leaders who inform trial protocols with lived experience.
What Sets Us Apart:
- 30+ years of frontline experience in neuromuscular conditions
- Support the development of MDA’s DMD Centers of Excellence and national care standards
- Trusted relationships with families from diagnosis through trial participation
- Developers of global education and care network support programs
- Ongoing presence at community events, youth camps, and advocacy forums
Our specialists collaborate with sites and sponsors, informing trial design, training site teams, and serving as liaisons to ensure the patient voice and needs are heard and reflected.
Team Member Spotlight:
Paula Orandash, Director of Patient Engagement
Paula Orandash, our Director of Patient Engagement, brings over 30 years of experience in patient advocacy, family support, and clinical care.
Paula has extensive experience in patient advocacy and clinical engagement, including a long tenure at the Muscular Dystrophy Association (MDA), where she supported patients across the care continuum—from diagnosis to clinical trial access. Most recently, she served as a Global Patient Engagement Liaison at a mid-size pharmaceutical company, working closely with patient advocacy groups to advance clinical trial initiatives. Her expertise spans patient-centered education, recruitment and retention, community engagement, and integrating the patient voice into research. Throughout her career, Paula has been dedicated to bridging patients, researchers, and industry to drive successful clinical trial outcomes.
Her unique blend of experience from both the advocacy and drug development sides makes her a powerful patient-centric leader, deeply connected to the communities she serves.
Operational Excellence with Real-World Trial Impact
Our delivery model strikes a balance between speed and precision, supported by agile systems and a global investigator network. We simplify complexity and reduce risk while maintaining the highest standards of scientific and ethical integrity.
Operational Highlights:
- First-patient-in achieved within 90 days across five countries
- Dedicated logistics teams with rare disease and pediatric trial experience
- Real-time country-level insights via our Strategic Solutions & Patient Centricity (SSPC) team
- Seamless vendor coordination and oversight
- Built-in risk mitigation strategies customized for neuromuscular trials
Accelerated Start-Up Through Regulatory Mastery
Our regulatory experts support submissions worldwide. In the EU, for example, our specialists guide sponsors through the requirements of the Clinical Trial Regulation (CTR) and the Clinical Trials Information System (CTIS) with speed and confidence.
Our EU Regulatory Performance:
- 18 successful submissions across Phases 1 to 4
- 100 percent on-time delivery and 12-day turnaround for all Requests For Information
- SOPs aligned with EMA standards; CTIS sandbox-trained teams
- Bilingual contract templates and streamlined negotiation frameworks
- Local regulatory leads manage end-to-end activation across countries
We monitor the EU portal daily, pre-plan translations, and move fast to prevent costly delays.
Overcoming Barriers to Enrollment and Retention
Neuromuscular disorder clinical trials often face significant hurdles—ranging from low patient prevalence to complex protocols and challenging endpoints.
Low Prevalance
Identifying and reaching eligible patients is one of the greatest challenges in neuromuscular trials. Ergomed deploys precision outreach and leverages global site networks to accelerate recruitment timelines. Our team activates high-performing, pre-qualified sites with relevant experience and access to rare patient populations—helping sponsors achieve enrollment goals faster.
Cognitive/Functional Barriers
Patients with neuromuscular conditions often face communication difficulties or physical limitations. Ergomed develops tailored communication approaches and integrates care network support tools that ensure accessibility, foster trust, and enable continued participation across diverse patient groups.
Trial Burden
Frequent site visits and complex procedures can discourage enrollment and affect retention. Ergomed minimizes burden through travel coordination, remote visit options, home nursing integration, and flexible scheduling models. Our personalized retention plans are designed to adapt to each patient’s capabilities and preferences.
Complex Modalities
Advanced therapies such as gene therapy and biomarker-driven interventions require specialized trial infrastructure. Ergomed brings in-depth expertise in cell and gene therapy trials, ensuring robust biomarker collection, real-time data capture, and streamlined logistics for high-complexity modalities.
Regulatory Hurdles
Navigating global regulatory frameworks can be daunting—especially in rare and progressive disorders. Ergomed’s in-country regulatory experts offer hands-on guidance for successful submissions, accelerated approvals, and ongoing compliance, ensuring studies stay on track from startup to closeout.
Subtle Endpoints
In neuromuscular research, endpoints can be nuanced and challenging to quantify. Ergomed collaborates closely with sponsors and regulators to develop sensitive, patient-relevant outcome measures that are both scientifically sound and regulatory-aligned. From digital biomarkers to patient-reported outcomes, we design endpoints that resonate.
Built for Real-World Success
We design every neuromuscular trial with real-world implementation in mind, ensuring clinical success and long-term impact for patients, care network, and sponsors alike.
How We Build Trials for Success:
- Data-driven feasibility and landscape assessments
- Strategic engagement with KOLs and patient advocacy organizations
- Cross-trial knowledge sharing to adapt and optimize in real time
- Longitudinal planning for post-marketing safety and RWE generation
Partner with Ergomed for Your Next Neuromuscular Study
From early-phase studies to global regulatory submissions, Ergomed delivers excellence in neuromuscular and neurodegenerative research. Whether you’re advancing gene therapies, SMA treatments, or ALS solutions, we bring agility, compassion, and clinical precision to your development journey.