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Samuel H. Hughes’ Vital Role in SMA Research – St. Jude Children’s Research Hospital

St. Jude Children's Research Hospital/LinkedIn

Aug 30, 2025, 15:58

Samuel H. Hughes’ Vital Role in SMA Research – St. Jude Children’s Research Hospital

St. Jude Children’s Research Hospital shared a post on LinkedIn:

“During Spinal Muscular Atrophy (SMA) Awareness Month, we highlight the impactful work of Samuel H. Hughes, MBA, operations manager for the Center for Experimental Neurotherapeutics (CENT), part of the St. Jude Pediatric Translational Neuroscience Initiative. Hughes’s dedication to translational research for ultrarare diseases, including SMA, exemplifies the commitment of St. Jude to advancing care for children facing life-threatening conditions.

Hughes played a pivotal role in a groundbreaking clinical trial led by Richard Finkel, MD, CENT director. This trial explored the potential of prenatal treatment for SMA, a disorder that, if left untreated, is often fatal. Recognized by TIME 100 Health for its promising results, the trial involved the administration of risdiplam to an expectant mother eight weeks before delivery, followed by continued treatment and careful monitoring after birth.

Hughes’s expertise in clinical research and logistical planning was crucial throughout the trial. From navigating complex regulatory requirements to ensuring timely collection and shipment of critical blood samples, his work enabled clinicians to accurately measure drug levels and adjust treatment protocols.

The collaborative effort extended well beyond St. Jude, involving regulatory agencies, pharmaceutical partners and care teams across multiple locations. Hughes reflected, “Everybody knew they had to move quickly; they understood the need and the potential impact of the study. It really took a village to make it all happen.”

The results of this trial demonstrate that prenatal treatment may be a promising approach for SMA. The child treated through this protocol, March Hennings, recently celebrated her third birthday and has no measurable signs of SMA, underscoring the potential impact of such research on future generations.”

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