Case Studies

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

Alex CheesmanMay 12, 2026

Study Overview 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…

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

Alex CheesmanApril 27, 2026

Study Overview 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…

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

The Challenge A global biopharmaceutical sponsor partnered with mdgroup to deliver a rare disease study focused on cerebral adrenoleukodystrophy (CALD). This rare manifestation of X-linked adrenoleukodystrophy (ALD) is a devastating…

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…

Innovating Pediatric Clinical Trials: Enabling Participation of Children with Complex Needs

Alex CheesmanMay 15, 2024

The Challenge mdgroup supported a groundbreaking initiative in pediatric decentralized clinical trials, focusing on children with SCN2A-DEE and SCN8A-DEE, which causes a range of neurological conditions, including severe early-onset epilepsies,…

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