Skip to Content

Rare and Urgent Diseases

There is nothing like rare disease research

We meet the challenges of rare disease studies head on – aligning the patient journey with our partners’ goals – building a robust strategy to move these programs forward.

Marc Gas Ph.D., Associate Director, Program Development

Every clinical trial is a challenge, no matter the therapeutic area. But trials in rare and urgent diseases are especially difficult due to the sparsity of the patient population, knowledge gaps in the research community, and a lack of study funding, among other difficulties.

These obstacles are why rare disease trials are

60% more likely

than other trials to face a major delay.

Speed and agility make the difference

Rare Disease

Speed and agility make the difference

The good news: Rare and urgent disease clinical trials share a common set of challenges. At Biorasi, we set out to solve those challenges and deliver successful rare disease clinical trials on time.

Rare Disease

Fast, responsive, and flexible

Rare Disease

Fast, responsive, and flexible

Over nearly 20 years, Biorasi has meticulously built a team and operating model with the capability to keep your study moving rapidly. And nowhere is that distinction more important than in rare and urgent diseases.

Rare Disease

Our rare and urgent disease experience

DERMATOLOGY RARE DISEASES

  • Generalized Pustular Psoriasis (GPP)
  • Palmoplantar Pustulosis (PPP)
  • Xeroderma Pigmentosum (XP)

GASTROENTEROLOGY RARE DISEASES

  • Eosinophilic Esophagitis (EoE)

GENETIC RARE DISEASES

  • Mucopolysaccharidosis Type II (MPS II)

HEMATOLOGICAL RARE DISEASES

  • Hereditary Hemorrhagic Telangiectasia (HHT)
  • Paroxysmal Nocturnal Hemoglobinuria (PNH)
  • Thalassemia

INFECTIOUS RARE DISEASES

  • Hepatitis D Virus

NEPHROLOGY RARE DISEASES

  • Calciphylaxis/Calcific Uremic Arteriolopathy (CUA)
  • Complement 3 Glomerulopathy (C3G)
  • Focal Segmental Glomerulosclerosis (FSGS)
  • Immunoglobulin A (IgA) Nephropathy
  • Lupus Nephritis (LN)

NEUROLOGY RARE DISEASES

  • Amyotrophic Lateral Sclerosis (ALS)
  • Friedreich’s Ataxia (FRDA)
  • Multiple Sclerosis (MS)

RARE CANCERS

  • Biliary Tract Cancer
  • Glioblastoma (GBM)
  • Multiple Myeloma (MM)
  • Renal Cell Carcinoma (RCC)

OPHTHALMOLOGY RARE DISEASES

  • Noninfectious Uveitis (NIU)
  • Pediatric Cataracts

RESPIRATORY RARE DISEASES

  • Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
  • Nontuberculosis Mycobacterium Infection (NTM)
  • Cystic Fibrosis (CF)
  • Idiopathic Pulmonary Fibrosis (IPF)

URGENT DISEASES

  • COVID-19