Rare Disease
CRO Services
Rare disease studies are high-stakes and high-complexity environments
Rare disease trials require a partner that can think quickly and creatively to solve unique problems. Across the spectrum of rare and orphan diseases, including ultra-rare indications, Precision for Medicine excels at providing rarefied solutions to solve the most complex of challenges.
A global CRO researching the depths of rare diseases
The clinical trials of rare and orphan diseases come with urgency and responsibility to extract the most meaningful data from every patient interaction and sample. Researching the depths of rare diseases often requires breaking new ground and pioneering clinical research in diseases yet to be investigated. Addressing the unique challenges of rare studies requires an agile and creative CRO capable of “Rarefied Thinking.”
Explore Precision’s specialized clinical development and commercialization capabilities for rare diseases:

Patients
Specialty Labs
Clinical Operations
Sites
Biostats
Biostats
Data Mgmt.
Data Management
Reg. Affairs
Reg. Affairs
HEOR
HEOR
Biomarker DM
Biomarker DM
Samples
Samples
Manu- facturing
Manufacturing

A rare disease CRO making a difference for more than a decade
Charting new territories in ultra-rare indications, pioneering orphan disease research, and understating populations via natural history studies—our experience spans more than 190 projects across 90+ rare indications.
- A1ATD
- Age-related Macular Degeneration
- Alport Syndrome
- Amyotrophic Lateral Sclerosis
- Autism Spectrum Disorders
- Dravet Syndrome
- Duchenne Muscular Dystrophy
- Fabry Disease
- Fragile X Syndrome
- Friedrich’s ataxia
- Gaucher Disease
- HAE
- Hereditary Inclusion Body Myopathy
- Multiple Sclerosis
- Neuro-autoimmune
- Neuromuscular Disorder
- Neuro-Ophthalmology
- Niemann-Pick Type C1 Disease
- PAH
- PNH
- Pompe Disease
- Prader Willi Syndrome
- Rare Genetic Syndromes
- Retinal vein occlusion
- Rett Syndrome
- Stargardt’s Disease
- Uveitis
- X-Linked Myotubular Myopathy
Rare disease CRO case studies
Experience matters in rare research Precision’s dedicated staff consistently delivers the creativity, collaboration, and agility necessary to surmount the unique challenges confronted when conducting clinical trials in rare diseases.
Indication:
Alport Syndrome
Services Provided:
Full-Service
Study Design(s):
Natural History & Phase II Program
Patient Population:
Child to Adult
Indication:
Rett Syndrome
Services Provided:
Project Management, Clinical Monitoring, Medical Monitoring, Site Start-up, and Management
Study Design(s):
Phase III pivotal, Open-Label Extension, Long term exposure, and Pediatric Open Label
Patient Population:
2 to 20 years old
Indication:
Inherited Metabolic Disorder
Services Provided:
Biostatistical Consulting on Efficacy, Endpoint Consideration for Approval

Specialty assays and laboratory solutions for rare research
Precision scientists know how to build creative solutions that holistically support the entirety of a rare disease clinical development project. Their support, together with our proprietary technologies, can help you overcome complex developmental and clinical study challenges:
- Epiontis ID for immune cell phenotyping
- ApoStream for isolating circulating tumor cells (CTCs)
- QuartzBio for integrating, analyzing and generating insight from diverse clinical and biomarker data
Work directly with the innovators who developed these technologies to find the right approach for your unique study requirements and endpoints.

Precision Convergence, the combined power of trials, labs and data sciences to drive faster clinical development
What set us apart is the way we integrate clinical trial execution with deep scientific knowledge, laboratory expertise, and advanced data sciences. This is Precision Convergence: maximizing insights into patient biology and accelerating the pace of scientific discovery and approval.