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Patient Story

Therapy Fit for a King
 Therapy Fit for a King 1024 588 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES
King Robinson - a young patient at Nationwide Children's - smiling and standing next to a window inside the hospital

Marsha Marshall decided to raise King Robinson when he was just a baby at the request of his mother, Tameka Robinson, who was suffering from complications of sickle cell disease. It wasn’t long before King was diagnosed as well. For a woman in her late 50s, it was a lot to manage, especially after her…

Mutation-Specific Therapy Brings New Hope for Patient With Duchenne Muscular Dystrophy
 Mutation-Specific Therapy Brings New Hope for Patient With Duchenne Muscular Dystrophy 1024 484 Abbie Miller
By: Abbie Miller
A man with Duchenne muscular dystrophy sits in a wheelchair between two family members

Justin Nicholoff is the first patient at Nationwide Children’s to receive del-zota through a managed access program. Since research into exon skipping therapies for Duchenne muscular dystrophy (DMD) took off about 15 years ago, Justin Nicholoff has waited for a therapy designed for his specific genetic mutation. In June, his wait ended as he became…

Nationwide Children’s Hospital Completes One of the World’s First Prenatal Treatments for Spinal Muscular Atrophy
 Nationwide Children’s Hospital Completes One of the World’s First Prenatal Treatments for Spinal Muscular Atrophy 1024 600 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES
Baby Bryson laughing at a check up following treatment for SMA.

Investigational third-trimester, transplacental “bridging” therapy with risdiplam — followed by rapid postnatal treatment — aims to protect motor neurons during a critical window before and immediately after birth.  When Stacy Auker found out she was pregnant with her third child, she knew a diagnosis of spinal muscular atrophy (SMA) was a possibility.  “We have relatives who have been affected, so I…

Setting the Stage the Next Era of Gene Therapy for Ultrarare Disease
 Setting the Stage the Next Era of Gene Therapy for Ultrarare Disease 1024 576 Abbie Miller
By: Abbie Miller

Researchers at Nationwide Children’s Hospital recently administered a novel gene therapy targeting SLC6A1, advancing precision medicine for children who previously could not be treated. Maxwell, age 8, made history this fall, when he received a bespoke gene therapy targeting his ultrarare disease. This wasn’t the first time he worked with the team at Nationwide Children’s…

Meet Prof Eileen Africa: Fulbright Scholar and Sports Medicine Expert
 Meet Prof Eileen Africa: Fulbright Scholar and Sports Medicine Expert 1024 683 Abbie Miller
By: Abbie Miller

Prof Eileen Africa, an associate professor in the Division of Movement Science and Exercise Therapy in the Department of Exercise, Sport, and Lifestyle Medicine at Stellenbosch University in South Africa and a Fulbright scholar, recently visited Nationwide Children’s to work with Dr. James MacDonald and the Sports Medicine Team conducting research about the effectiveness of…

Meet Antonio Cabrera, MD, Chief of Cardiology at Nationwide Children’s Hospital
 Meet Antonio Cabrera, MD, Chief of Cardiology at Nationwide Children’s Hospital 150 150 Abbie Miller
By: Abbie Miller

Antonio Cabrera, MD, FAAP, FAHA, FACC, FHFSA, is the division chief of cardiology and co-director of the Heart Center. He is a professor of pediatrics at The Ohio State University and currently holds the Nationwide George H. Dunlap Endowed Chair in Pediatric Cardiology. Previously, Dr. Cabrera served as the L. George Veasy Presidential Professor at…

Patient Perspective: Living With Weight-Based Stigma
 Patient Perspective: Living With Weight-Based Stigma 1024 376 Alaina London
By: Alaina London

In 2019, 15-year-old Paige watched her father die from a disease that was consistently ignored, and his symptoms repeatedly blamed on his weight. He had stage 4 cancer by the time he was properly diagnosed. Shortly after his death, her mother had a 40-pound cyst removed, a surgery that she and other doctors believe could…

Optimizing Prenatal and Neonatal Care for Infants With Treatable Rare Diseases
 Optimizing Prenatal and Neonatal Care for Infants With Treatable Rare Diseases 1024 619 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

As new treatments emerge and diagnostics improve, earlier interventions offer infants with rare metabolic and neurodegenerative conditions a future wildly different than ever before. Not long ago, a diagnosis of molybdenum cofactor deficiency (MoCD) type A meant death before kindergarten. Since the FDA approval of NULIBRY® (fosdenopterin) in 2021, however, children diagnosed with MoCD type…

The First 3 Years With PBS: Bryson’s Story
 The First 3 Years With PBS: Bryson’s Story 953 269 Wendy Margolin
By: Wendy Margolin

The first time Andrea Williams met her new baby was four days after his birth. Baby Bryson was born with the rare congenital disorder, prune belly syndrome (PBS), and was rushed to the Nationwide Children’s Hospital NICU immediately after birth. His enlarged belly meant he was delivered by C-section at 37 weeks, and it wasn’t…

Building Hope, Recovery, and Life Beyond Substance Use Disorder
 Building Hope, Recovery, and Life Beyond Substance Use Disorder 1024 702 Abbie Miller
By: Abbie Miller

Five years ago, Pediatrics Nationwide dedicated its fall issue to a comprehensive look at the effects of the opioid crisis on children and families. At that time, Sarah Parker was working hard to stay sober, recovering from the very crisis holding the nation’s attention. Parker grew up in Chillicothe, Ohio, halfway between Portsmouth, Ohio, the…

Fetal Surgery for Spina Bifida: Supporting A Bright Future Closer to Home
 Fetal Surgery for Spina Bifida: Supporting A Bright Future Closer to Home 150 150 Alaina London
By: Alaina London

During her 20-week ultrasound appointment, Brittany Badenhop learned her baby had spina bifida, a term used to describe many disorders that occur during the development of the central nervous system. Spina bifida occurs very early on in a pregnancy as the embryo forms. Brittany and her husband Jared learned that their baby’s spine was not…

Robotic Approach to Rare Case Leads to Best Possible Outcome
 Robotic Approach to Rare Case Leads to Best Possible Outcome 1024 683 Emily Siebenmorgen
By: Emily Siebenmorgen

A rare tumor in a child from Greece is resolved by robotic spleen-sparing distal pancreatectomy. Hospital visits can feel like a foreign experience for anyone, especially so when your hospital is actually in another country. But eight-year-old Evangelia felt especially at home after traveling to Nationwide Children’s Hospital from Greece for her care. Evangelia’s first…

The Impact of Genomic Medicine: Carter’s Story
 The Impact of Genomic Medicine: Carter’s Story 1024 685 Natalie Wilson
By: Natalie Wilson

In 2013, at just 1 year old, Carter Daggett was diagnosed with a rare brain tumor. Now, in 2021, at 8 years old, he celebrated three years cancer free — thanks to his clinical teams and cutting-edge research by the team at the Steve and Cindy Rasmussen Institute for Genomic Medicine at Nationwide Children’s Hospital.…