Tailor-made: The world’s first personalized gene-editing treatment Baby KJ was a pioneer before he was even a year old. Born in 2024 with a rare, life-threatening genetic mutation, Nicole and Kyle Muldoon’s son is the world’s first patient to receive successful personalized gene editing therapy. Doctors at the Children’s Hospital of Philadelphia and Penn Medicine collaborated to create a unique drug just for KJ, to treat his disorder. Now, a busy toddler and youngest brother, KJ is a piece of medical history and he just started to walk.

KJ is the world’s 1st recipient of a personalized gene therapy.

In @CNBC Cures’ latest feature, his parents share their journey after his diagnosis at just 2 days old—and the treatment that helped save his life: ms.spr.ly/63322QC4OG.

At Children’s Hospital of Philadelphia, we are heartbroken by the loss of the three construction workers in the structural collapse of the garage under construction in Grays Ferry. We extend our deepest condolences to their families, loved ones, and the many dedicated members of Ironworkers Local Union 401 and the Philadelphia Building and Construction Trades Council. They have long played an important role in building facilities that make our mission possible. The loss of life is deeply felt here as we are constantly reminded how precious and fragile life is.

We are grateful to the first responders and to Mayor Cherelle L. Parker and the City of Philadelphia for their tireless efforts, exploring every possible rescue option and ensuring the safety of everyone impacted, including nearby neighbors and businesses. We recognize the impact of this tragic incident on them, and we will collaborate with the City to support their needs. We know there are questions about the future of the site, but CHOP’s current priority is to fully cooperate with government agencies as they seek to investigate the cause of this tragic incident.

A statement from Children's Hospital of Philadelphia.

Period poverty can prevent people from attending school, work or job interviews. The Period Project provides period packs with supplies for a full cycle, promoting health, dignity and opportunity.

Period poverty can prevent people from attending school, work or job interviews. The Period Project provides period packs with supplies for a full cycle, promoting health, dignity and opportunity.

Learn more in our Community Impact Report: ms.spr.ly/63326tmRq8.

Pediatric Dentistry and Sleep Disorders (Episode 205) In this episode, Andrew Cohen, DMD, who is board certified in dental sleep medicine and a comprehensive restorative dentist, explains how anatomy, behavior, and overall health impacts sleep-related breathing. We discuss common issues like mouth-breathing, teeth grinding, thumb-sucking, and extended pacifier use and how these impact dental health and sleep. For more information, visit the CHOP Sleep Center.   Opinions expressed in this episode are those of Andrew Cohen, DMD, and not necessarily representative of Children's Hospital of Philadelphia.

In this Primary Care Perspectives episode, Dr. Katie Lockwood speaks with Dr. Andrew Cohen about how a child's anatomy, behavior, and overall health can impact sleep-related breathing. Listen below or wherever you stream your podcasts.

Bridging Biomaterials Research and Clinical Care: Advancements in Sustainable Healthcare Craniosynostosis (CS) is a congenital condition characterized by the premature fusion of cranial sutures due to abnormal developmental pathways (Governale et al., 2015). This fusion can lead to increased intracranial pressure, cognitive impairment, and visible skull deformities. Current treatments include helmet therapy and minimally invasive surgical procedures. However, re-craniosynostosis can occur, limiting the long-term effectiveness of surgery. In addition, these procedures are associated with significant financial costs and carry risks such as substantial blood loss. Through the Princeton IIP program, I had the opportunity to research sustainable alternatives for CS treatment.

Read the latest Princeton Medical Review article by a Princeton University student, Olivia Bell, who shared her experience shadowing CHOP neurosurgeon, Dr. Tracy Flanders, and witnessing the compassion and communication skills required in this field.

Daniel P. Kelly Honored as Distinguished Scientist by the American Heart Association Daniel P. Kelly, MD, CHOP's Cardiovascular Institute Director, won AHA's 2025 Distinguished Scientist Award for cardiovascular research. Children’s Hospital of Philadelphia (CHOP) is pleased to announce that Daniel P. Kelly, MD, Director of the Cardiovascular Institutes at CHOP and the Perelman School of Medicine at the University of Pennsylvania and a renowned researcher in the field of cardiovascular and metabolic diseases, was presented with the prestigious 2025 Distinguished Scientist Award by the American Heart Association (AHA) at the organization’s Scientific Sessions in New Orleans on Saturday, November 8th. The AHA’s Distinguished Scientist award, established in 2003, celebrates members who have made extraordinary contributions to research in cardiovascular, stroke, and brain health.“I am deeply honored to receive the 2025 Distinguished Scientist award," said Kelly. “Support from the AHA was crucial in launching my research, allowing me to make meaningful contributions to pediatric heart health by fostering connections with fellow scientists and collaborators.”Kelly's research journey began with a focus on rare genetic metabolic disorders that lead to cardiomyopathy and sudden death in children. This evolved into a broader mission to uncover the metabolic roots of heart disease. Among his groundbreaking work, Kelly identified genetic causes affecting mitochondrial fat metabolism, which led to practical newborn screening tests.His research has transitioned from fundamental discoveries to developing new therapies that address metabolic imbalances in heart failure. One of these therapies progressed to human clinical trials, marking a significant milestone in potential heart disease treatments.Kelly has received numerous accolades throughout his career. He is a member of the American Society for Clinical Investigation and the Association of American Physicians, where he served as President in 2022. His previous honors from the AHA include the 1995 Established Investigator Award, the 2008 Basic Cardiovascular Science Council Distinguished Achievement Award, and the 2009 Basic Research Prize.

Dr. Daniel Kelly, Director of the Cardiovascular Institutes at CHOP and @pennmedicine.bsky.social, received the 2025 Distinguished Scientist Award from the @American_Heart. His groundbreaking work in cardiovascular and metabolic diseases continues to inspire.

NASPGHAN 2025 Mousa Kapavarapu

Our final #NASPGHAN Meet the Experts session this year features Drs. Hayat Mousa and Prasanna Kapavarapu discussing our motility fellowship at Tower Exhibit #7-8. #NASPGHAN25

NASPGHAN 2025 Pai

Our next #NASPGHAN Meet the Experts session features Dr. Nikhil Pai discussing our nutrition fellowship. The session is in Tower Exhibit #7-8. #NASPGHAN25

NASPGHAN 2025 Albenberg

Dr. Lindsey G. Albenberg will be discussing our advanced IBD fellowship in our next #NASPGHAN Meet the Experts session in Tower Exhibit #7-8. #NASPGHAN25

NASPGHAN 2025 Ryan

Good morning #NASPGHAN attendees! We have another great day of Meet the Experts sessions planned for today. Dr. Matthew J. Ryan will be discussing our aerodigestive medicine fellowship as well as transnasal endoscopy in Tower Exhibit #7-8. #NASPGHAN25

NASPGHAN 2025 Kamath

Our next #NASPGHAN Meet the Experts session features Dr. Binita Kamath. She will be discussing all things GI, hepatology and nutrition beginning in Tower Exhibit #7-8. #NASPGHAN25

NASPGHAN 2025 Brownell

Dr. Jefferson Brownell will be discussing pancreatic and lymphatic at our next #NASPGHAN Meet the Experts session in Tower Exhibit #7-8. #NASPGHAN25

NASPGHAN 2025 Maxwell and Conrad

Our next #NASPGHAN Meet the Experts session features Drs. Elizabeth Clabby Maxwell and Marie Abraham Conrad discussing our fellowship program and very-early onset inflammatory bowel disease. This session is in Tower Exhibit #7-8. #NASPGHAN25

Maria Mascarenhas at NASPGHAN 25

Our Meet the Experts sessions begin today at #NASPGHAN! Dr. Maria Mascarenhas will be discussing culinary medicine and integrative health today in Tower Exhibit #7-8. #NASPGHAN25

CHOP at NASPGHAN 25

Are you attending #NASPGHAN 2025 in Chicago? Get ready to connect with our experts on November 7th and 8th at Tower Exhibit #7-8. Stay tuned to see upcoming expert talks. #NASPGHAN25

ASHG Elizabeth Bhoj

Dr. Elizabeth Bhoj recently received the @geneticsociety.bsky.social Early Career Award for her extensive work in translational genetics to improve diagnosis and treatment of novel human syndromes. #ashg2025

Frest fruit and vegetables with hands forming a heart

We understand that the government shutdown is impacting vital support of SNAP and WIC effective 11/1 for our families and community members. Access to healthy food is crucial. If you need assistance, check out local resources here: ms.spr.ly/63328tEcIm.

Football Protective Equipment and Concussion Risk (Episode 203) Podcast Episode · Primary Care Perspectives · 10/28/2025 · 32m

In this Primary Care Perspectives episode, two of our Center for Injury Research & Prevention experts discuss how parents, coaches & trainers can help monitor for a concussion and improve recovery.

🎧 Listen below or wherever you stream your podcasts.

CHOP’s Center for Data Driven Discovery in Biomedicine finds AI in Pediatric Neuro-Oncology Surpasses Manual Measures

Researchers in our Center for Data Driven Discovery in Biomedicine have released new guidance in @thelancet.com to help responsibly implement artificial intelligence in the assessment and treatment of brain tumors in children. Learn more: ms.spr.ly/63321tD7FH.

CHOP at AACAP

What a great week at the AACAP Annual Meeting! Our team of experts enjoyed meeting #AACAP25 attendees. Let’s work together to bring the village to the children, families, and communities we serve.

CHOP at AACAP

If you are at #AACAP25, stop by our booth #440 to meet our team. Our clinicians are speaking & presenting throughout the week. Learn more: ms.spr.ly/63324shWX8.

CHOP at AACAP

At #AACAP25 and interested in learning more about our mental health services and training programs? Stop by @AACAP booth #440 to meet some members of our team. Learn more: ms.spr.ly/63322sCSNj.

CHOP at AACAP

At #AACAP25? Stop by & meet our experts at our @AACAP booth #440. Our team will be speaking throughout the conference & you will not want to miss valuable insights from our leaders. Learn more: ms.spr.ly/63327sCS2c.

CHOP at AACAP

Clinicians and experts from our Department of Child & Adolescent Psychiatry & Behavioral Sciences are presenting at @AACAP this week. Stop by booth #440 to meet our team. Check out the presenters & topics: ms.spr.ly/63322sCRVe.

KJ and Nicole with Drs. Kiran Musunuru and Rebecca Ahrens-Nicklas

Drs. Kiran Musunuru and Rebecca Ahrens-Nicklas

Life Sciences Pennsylvania hosted a special panel discussing the CRISPR technology used to treat KJ Muldoon for his ultra rare metabolic disorder. Our docs and KJ's mother, Nicole, shared the story from the perspectives of the scientists and the caregivers.

Oral Immunotherapy (Episode 202) Podcast Episode · Primary Care Perspectives · 10/08/2025 · 29m

Could eating an allergen help you overcome an allergy? In this Primary Care Perspectives episode, learn more about a promising treatment option: oral immunotherapy. 🎧 Listen below or wherever you stream your podcasts.

CHOP at AACAP

Our Psychiatrist-in-Chief & @AACAP President, Dr. Tami Benton, will be participating in speaking engagements throughout the conference & will be stopping by our booth #440. Find out more about important mental health topics our team will be covering: ms.spr.ly/63325s7irJ. #AACAP25

Time to celebrate! We're proud to once again be ranked one of the nation’s best pediatric hospitals by U.S. News & World Report! Join us in congratulating our first-class teams who continue to make breakthroughs happen for kids everywhere. 📣

ms.spr.ly/63320sLHzp

CHOP AAP 2025 Thanks

Thanks to everyone at #AAP2025 who stopped by our booth and chatted with our range of experts at the conference! We look forward to seeing many of you again next year in San Diego.

CHOP AAP 2025 Monday

Today is your last day at #AAP2025 to meet some of our experts at booth #1902! We hope to see you there.