We KNOW RARE. WE ARE RARE.

Our Experience in Rare Diseases

 Acute Hepatic Porphyria

Alport Syndrome

Amyotrophic Lateral Scerosis

Aatypical Hemolytic Uremic Syndrome (aHUS)

C3 Glomerulopathy hATTR Amyloidosis

Chronic Lymphocytic Leukemia

Chronic Myelogenous Leukemia

Common Variable Immunodeficiency 

Congenital TTP

Cystic Fibrosis 

Duchenne Muscular Dystrophy

Fabry Disease

Familial Chylomicronemia Syndrome    

Gaucher Disease

Hemophilia A

Hemophilia B

Homozygous Familial Hypercholesterolemia

Hypoparathyroidism

Lipodystrophy

Mantle Cell lymphoma

MPS I, II (Mucopolysaccharidosis )

Myasthenia Graves

Paroxysmal Nocturnal Hemoglobinuria (PNH) 

Polycythemia Vera

Pompe Disease

Primary Immunodeficiency

Pyruvate Kinase Deficiency 

Sickle Cell Disease

Spinal Muscular Atrophy

Tuberous Sclerosis 

Von Willebrand disease

WHIM Syndrome

Core Principles Drive All We Do

  

scientific 

a deep understanding of the science across the team is at the root of every thing we do


entrepreneurial 

our spirit embodies creativity and out-of-the-box thinking and our responsiveness and flexibility allow you to be nimble


global 

we embrace global vision and local impact with streamlined global and US launches under our belts and resources across the globe


value-driven 

we build a team of experts for your specific project and only charge you for what we do

 

We are rare


We know rare. we are rare.