RARE DISEASE & ORPHAN DRUG

Broad Experience in Rare and Orphan Diseases

Avance Clinical has successfully completed over 60 trials in rare and orphan disease populations, globally in the past five years. Our experience is demonstrated by our continued ability to manage global patient trials in rare indications, contributing to innovative trial design and ensuring a patient-centric approach with the relevant advocacy groups involved.

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22q11.2 Deletion Syndrome

Acromegaly

Acute Respiratory Disease Syndrome

Alpha-1 Antitrypsin Deficiency

Cystic fibrosis

Duchenne Muscular Dystrophy (DMD)

Erythropoietic Protoporphyria (EPP)

Fibrodysplasia ossificans progressiva (FOP)

Fragile X Syndrome

Frontotemporal Dementia (FTD)

GNE Myopathy

Gonadotropin-Dependent Precocious Puberty

Hemochromatosis

Hereditary Angioedema

Hypoxic-ischemic encephalopathy

Idiopathic pulmonary fibrosis

Myasthenia Gravis

Myotonic Dystrophy

Narcolepsy

Netherton Disease

Palmoplantar Pustulosis

Retinitis pigmentosa

Rett Syndrome

Segmental Glomerulosclerosis

Unexplained Anemia of Aging (UAA)

Usher Syndrome

Vanishing White Matter

Avance Clinical’s Expertise in Rare Diseases
Download our Guide Today.

Avance Clinical's Expertise in Rare Diseases
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