Case Studies

Supporting At-Home Treatment for a Phase 4 Immunodeficiency Trial

Alex CheesmanAugust 4, 2026

Discover how mdgroup delivered 64 in-home visits across a Phase 4 primary immunodeficiency trial, enabling home-based treatment, zero missed visits and high-quality clinical data through Decentralized Clinical Services.

Delivering mdgroup’s First Medical Air Transport for a Pediatric Rare Disease Trial

Alex CheesmanMay 12, 2026

Supporting a global clinical study in ultra-rare metabolic disorders, mdgroup delivered its first-ever medical air transport solution, enabling a one-year-old patient and their family to safely access a clinical trial…

Delivering Global Access to Life-Changing Gene Therapy for Children with Ultra-Rare Disease

Alex CheesmanApril 27, 2026

In 2019, mdgroup partnered with Rocket Pharmaceuticals to support a Phase I/II clinical study investigating a gene therapy for pediatric patients with severe Leukocyte Adhesion Deficiency-I (LAD-I), an ultra-rare, life-threatening…

Pediatric Global Gene Therapy Clinical Trial Support for AADC Deficiency

Alex CheesmanJanuary 28, 2026

This global gene therapy study required highly specialized, long-term patient support to enable participation for children with severe mobility and care needs across multiple regions. AADC deficiency is a rare, severe…

Enabling Global Participation in a Rare Disease Hybrid Study for Cerebral Adrenoleukodystrophy (CALD)

Alex CheesmanNovember 14, 2025

A global biopharmaceutical sponsor partnered with mdgroup to support a hybrid rare disease clinical trial, bringing together participants from around the world to evaluate a potential treatment for CALD. To…

Boy in a wheelchair looking back over his shoulder

Supporting Patients with Friedreich’s Ataxia: Seamless Travel and Reimbursement in a Global Study

Alex CheesmanJune 19, 2025

A leading global biopharmaceutical company contracted mdgroup to provide travel coordination and reimbursement support for patients participating in a long-term clinical trial for Friedreich’s ataxia (FRDA).

Website image_Case Study-20_Innovating Rare Disease Research with mobilehealth

Innovating Rare Disease Research with Decentralized Clinical Services – A Breakthrough for Danon Disease Patients

Alex CheesmanDecember 18, 2024

In a groundbreaking effort to make clinical trials more accessible for patients with rare diseases, mdgroup successfully conducted mobile health visits for two brothers in Tel Aviv, Israel, diagnosed with…

Bahrain Patient Engagement Milestone

Alex CheesmanAugust 14, 2024

The mdgroup team has hit another patient engagement milestone by assisting a patient from a small town in the Island country of Bahrain. This Island off the coast of West…

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