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Rare Neurological Disorders

Passionate, Dedicated Teams Committed to Rare Neurological Disease Research

The execution of any rare disease trial requires different skill sets. This is especially true with rare disorders affecting the central nervous system.

We attack these trial challenges head-on by embedding specialized and flexible development strategies that include a rigorous assessment of feasibility, robust training and supportive site network relationships, and collaborations with patient advocacy groups, patient concierge services and more.

Solutions for Neurodegenerative Clinical Trial Sites and Patient Access

• 100+ rare neurological trials
• Pediatric expertise
• Gene therapy experience
• Indication-specific site partnerships

Experience with a Diverse Array of Rare Neurological Indications:

  • Amyotrophic lateral sclerosis (ALS) 
  • Angelman syndrome 
  • Batten 
  • Cerebral palsy 
  • Cerebral cavernous malformations 
  • Cervical dystonia 
  • Chronic inflammatory demyelinating 
    polyneuropathy
  • Dravet syndrome 
  • Duchenne muscular dystrophy 
  • Epileptic encephalopathies  
  • Fabry disease 
  • Facioscapulohumeral muscular dystrophy,  
  • Frontotemporal dementia 
  • Fragile X syndrome
  • Gangliosidosis GM2 
  • Gaucher disease 
  • Huntington’s disease 
  • Lennox-Gastaut syndrome 
  • Lysosomal acid lipase deficiency 
  • Metachromatic leukodystrophy
  • Mitochondrial disease 
  • Mucopolysaccaridosis (I-III) 
  • Myasthenia gravis 
  • Multiple systems atrophy 
  • Neuromyelitis optica 
  • Prader-Willi 
  • Spinocerebellar ataxia 
  • Tardive dyskinesia

Ending Neuromuscular Disorders

PPD combines our rare disease, gene therapy and direct neuromuscular trial experience to help bring patients independence and freedom.

Lean more about our neuromuscular experience

Expertise in the Unique and Novel Aspects of Rare Neurological Research

As the pipeline of new rare neurological disease treatments expands, PPD is ready. We have expertise in understanding these complex diseases, meeting regulatory requirements, and measuring what is important to patients and families.

  • Gene therapy medical and operational expertise 
  • First-in-human single-ascending-dose (SAD) Phase Ib/IIa through Phase IV trials 
  • Natural histories, registries, endpoint development, and analysis 
  • Complex studies with multiple vendor requirements, such as biopsy, clinical outcome assessments (COAs), labs, biolabs, physiotherapy, MRI

Keeping the Patient at the Center of the Trial

We foster a collaborative environment that includes input from patients, providers and caregivers to understand the patients’ needs early in the process – before the study protocol has been finalized to help reduce their study burden. We keep patients top of mind through:

  • Expertise from our rare disease and pediatric center of excellence
  • Concierge services: payment reimbursement to patient burden reduction services, including call center, transportation management, home health care, televisits
  • Digital trial solutions
  • Relationships with patient advocacy groups and partnerships with indication-specific site networks
  • Patient perspective guidance to reduce burden