Case Studies Archive

Pediatric Global Gene Therapy Clinical Trial Support for AADC Deficiency

Posted | January 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…

Alex Cheesman

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

Posted | November 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…

Alex Cheesman

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Boy in a wheelchair looking back over his shoulder

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

Posted | June 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).

Alex Cheesman

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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

Posted | December 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…

Alex Cheesman

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Bahrain Patient Engagement Milestone

Posted | August 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…

Alex Cheesman

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

Posted | May 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,…

Alex Cheesman

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

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

Posted | April 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…

Alex Cheesman

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Overcoming Clinical Research Staffing Challenges

Posted | April 5, 2024

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

Alex Cheesman

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Changing Lives – Creating Remarkable Long-term Trial Experiences

Posted | April 27, 2023

Overcoming cultural, geographical, and financial barriers to facilitate a life-changing trial for one of the world’s rarest diseases The Challenge A very young patient had the opportunity to participate in…

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Case Studies Archive - mdgroup

Pediatric Global Gene Therapy Clinical Trial Support for AADC Deficiency

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

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

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

Bahrain Patient Engagement Milestone

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

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

Overcoming Clinical Research Staffing Challenges

Changing Lives – Creating Remarkable Long-term Trial Experiences

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Case Studies Archive - mdgroup

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