PPD Inc Main Menu Have a question? Contact us today! RARE NEUROLOGICAL DISORDERS Therapeutic Expertise Neuroscience Research 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. • 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 Legal Notices Site Map Modern Slavery Transparency Statement Privacy Policy ©2021 PPD Inc. All Rights Reserved. Contact Us LinkedIn Twitter Facebook Instagram YouTube Events By clicking “Accept All Cookies”, you agree to the storing of cookies on your device to enhance site navigation, analyze site usage, and assist in our marketing efforts. Accept All Cookies Cookies Settings