Rare Diseases and Orphan Drugs

Research in rare disease & orphan drugs requires incisive thinking, experience, & a gentle touch.

We share your passion for finding treatments for intractable disease states.

Understanding the exceptional challenges of research for rare disease and orphan drugs is essential in bringing treatments to the patients who need them. Fifty percent of rare diseases affect children, placing great burdens on families, while the intricacies of genetic research require highly specialized knowledge. Patient-centric trial designs are paramount.

Rare diseases can impact a single system of the body or several, making monitoring progression a challenge. Our Integrated Full-Service Rare Disease Solution leverages insights gathered through the conduct of 120+ rare disease trials to facilitate decision making and improve study design and execution.

From natural history studies to pivotal trials, our expertise includes complex clinical trial considerations such as pediatric patient populations, new biomarkers, surrogate endpoints, and the incorporation of patient perspectives and experiences, across a wide range of innovative medicines and advanced therapeutics in rare and ultra-rare diseases.


Therapeutic Experience You Can Depend On

For more than 20 years, Allucent has specialized in rare and orphan disease research. Our team has supported more than 120 clinical trials, including 16 product approvals, across a variety of novel therapy classes and precision medicine including gene therapies, GMOs, stem cell therapies, and mRNA and antisense therapies.

Rare Disease

  • Acromegaly (All Subsets)
  • Epilepsies (pediatric, adult; progressive, genetic, refractory…)
  • Autoimmune hemolytic anemia (CAD, PNH, WAD)
  • Blood clotting disorders (ITP, aHUS, TTP…)
  • Spasticity and Sialorrhea disorders (CP, MS, Stroke, Trauma…)
  • Movement disorders (Duchenne, CP, Ataxia, Huntington’s …)
  • Lysosomal storage disorders (different subtypes, details available upon request)
  • Gastrointestinal disorders (Short bowel…)
  • Pulmonary disorders (CF, PAH, Bronchiectasis..)
  • Cardiac disorders (ATTR-CM amyloidosis…)
  • Skin disorders (RDEB, DDEB)
  • Rare CNS disorders
  • Metabolic storage diseases
  • Infectious diseases
  • Cardiovascular diseases
  • Immunologic disorders

Rare Disease and Orphan Drug Development Expertise

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Oncology and Hematology

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Infectious Disease and Vaccine

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Cell and Gene Therapy

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Central Nervous System

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Autoimmune and Inflammation

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Allergy and Asthma

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