Case Studies

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 Paediatric Clinical Trials: Enabling Participation of Children with Complex Needs

Alex CheesmanMay 15, 2024

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

image_Case-Study-16_Amyotrophic-lateral-sclerosis-ALS

Facilitating a Large-scale Global Study on Amyotrophic Lateral Sclerosis (ALS)

Alex CheesmanApril 8, 2024

mdgroup is currently supporting a global study on the rare disease Amyotropic Lateral Sclerosis (ALS), a nervous system disease that affects nerve cells in the brain and spinal cord. We…

Overcoming Clinical Research Staffing Challenges

Alex CheesmanApril 5, 2024

A global CRO faced a daunting challenge in managing a clinical research site, known for its staffing shortages and difficult working conditions.

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