---
title: "Rare disease & orphan drug Services - Avance Clinical"
description: "Establishing partnerships with patient advocacy groups, key opinion leaders, and specialized centers can enhance access to patients and expertise in this sector."
canonical: "https://www.avancecro.com/therapeutic-areas/rare-disease-orphan-drug/"
generated: "2026-09-17T09:39:18+00:00"
---

# **[Rare Disease](https://www.avancecro.com/therapeutic-areas/rare-disease-orphan-drug/) & [Orphan Drug](https://www.avancecro.com/therapeutic-areas/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.

Discover Avance Clinical’s Expertise in Rare Diseases: Download Our Guide Today.

[Request a proposal](https://www.avancecro.com/request-a-proposal/)

![](https://www.avancecro.com/wp-content/uploads/2024/05/Cancer-Cells-under-microscope-aspect-ratio-1600-975-916x916.jpg)

## GET IN TOUCH WITH OUR **CLINICAL TRIAL EXPERTS**

Three decades of experience in biotech drug development

[Connect with us](https://www.avancecro.com/request-a-proposal/)

## Our **expertise**

[Request a proposal](https://www.avancecro.com/request-a-proposal/)

### 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.