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Jake Larose is a now healthy 13-year-old boy who experienced a severe medical journey after developing hemolytic uremic syndrome from eating a hamburger in March 2014. His condition rapidly progressed, leading to acute renal failure, peritonitis, bowel perforation, severe septic shock, and acute respiratory failure. Throughout his stay in the Pediatric Intensive Care Unit (PICU) at NewYork-Presbyterian Komansky Children’s Hospital, Jake received extensive multidisciplinary care and rehabilitation. Despite experiencing delirium and hallucinations during his ICU stay, Jake made a remarkable recovery and was eventually discharged home. Though he still requires ongoing medical care, nearly a decade later, Jake is doing well. 

Dr. Zachary Grinspan, Interim Division Chief of pediatric neurology at Weill Cornell Medicine and Director of the Pediatric Epilepsy Program, has been awarded an R01 from the National Institute of Neurological Disorders and Stroke for his study, “NS-PEACE Neonatal Seizures -Predicting Epilepsy and Assessing Comparative Effectiveness.” The study aims to improve the care of newborns who experience seizures and their subsequent outcomes and predict and prevent epilepsy in high-risk individuals.

Neonatal seizures occur once per 1000 live births and are associated with the subsequent development of epilepsy, cerebral palsy, and intellectual disability. This study will address three questions related to the management of acute symptomatic neonatal seizures. First, it will compare the effectiveness of two anti-seizure medications, levetiracetam and phenytoin/fosphenytoin, to determine which is the best second-line medication. Second, it will examine whether the use of certain medications, like oxcarbazepine, affects the risk of infantile spasms syndrome. Finally, it will validate a published epilepsy prediction rule to determine which infants will develop epilepsy after leaving the neonatal intensive care unit.  

Full article posted in WCM Newsroom

Dr. James C. Lo, an associate professor of medicine and Dr. Lisa G. Roth, an associate professor of pediatrics, both from Weill Cornell Medicine, were elected members of the American Society for Clinical Investigation (ASCI) for 2023.

Election into the ASCI, which represents excellence across the breadth of academic medicine, is a milestone for physician-scientists. The nonprofit medical honor society, which is comprised of more than 3,000 physician-scientists from all medical specialties, recognizes and supports the scientific efforts, educational needs and clinical aspirations of physician-scientists to improve the health of all people. 

Congratulations to Dr. Zoltan Antal, chief of the Division of Pediatric Endocrinology and director of the Pediatric Diabetes Program, on receiving the Weill Cornell Medicine Marie Metoyer, M.D. Award. The award is given to a current faculty member or Weill Cornell Medical College alumnus who epitomizes the legacy of Dr. Metoyer, the first Black woman to graduate from Weill Cornell Medicine who valued healing and community engagement. The award honors those who have gone above and beyond to serve underprivileged communities in an unorthodox manner.

Weill Cornell Medicine honored nearly a dozen faculty, students and staff who exemplify excellence in diversity, equity and inclusion for their commitment to fostering inclusivity in academic medicine on April 25th. Read more.

This article was originally posted in the NORD newsroom.

Washington, DC, May 4, 2023— Today, the National Organization for Rare Disorders (NORD) announced the addition of nine new Rare Disease Centers of Excellence, bringing the total number of medical centers in this network to 40 on the day of the 40th anniversary of NORD’s founding.

The nine new NORD Rare Disease Centers of Excellence announced today include:

Congratulations to Dr. Julia Buirkle on being awarded the 2023 CATCH grant by the American Academy of Pediatrics (AAP) in recognition of her outstanding work in child health.

The Community Access to Child Health (CATCH) program, offered by the AAP, supports pediatricians in their efforts to collaborate with communities and advance the health of all children. With the CATCH Grant, Dr. Buirkle will work closely with Team KiPOW!, a national nonprofit organization, to implement school wellness policy in underserved elementary/middle schools and help children reach their full physical, emotional, and academic potential through improved nutrition and physical activity.

As part of the project, Dr. Buirkle and her team at NewYork-Presbyterian/Weill Cornell Medical Center, known as “Team KiPOW! Cornell” will work with P.S. 083 Luis Munoz Rivera School in East Harlem in the form of an after-school program and study its impact on the health of participating students through collecting questionnaires and biometric data. This project also offers a valuable opportunity for medical students and residents to gain training and experience in nutrition and lifestyle counseling.

Katiana Garagozlo M.D. is a PGY-6 fellow in our Pediatric Pulmonology Program at NewYork-Presbyterian Komansky Children’s Hospital at Weill Cornell Medical Center. Dr. Garagozolo completed her residency at Broward Health Medical Center/Salah Foundation Children’s Hospital and medical school at the American University of the Caribbean School of Medicine.

Hi Dr. Garagozlo, will you share with us what life looks like for a third-year Pediatric Pulmonology fellow?

As a third-year fellow, our clinical responsibilities are reduced compared to that of the first-year fellow. Our time is spent focusing on completion of research projects and other scholarly work. We also act in the role of chief fellow, which includes administrative responsibilities such as creating the fellow on-call schedules, as well as assisting the program director in creating the didactic schedules.

This article was originally posted in Neurology Live Spotlight News

New interim results from the phase 2 open-label NEXUS (NCT04528706), 96-week study on leriglitazone (Minoryx Therapeutics), a peroxisome proliferator-activated receptor γ agonist, for pediatric cerebral adrenoleukodystrophy (cALD) showed that all evaluable patients were clinically stable and radiologically displayed disease arrest or lesion growth stabilization after 24 weeks of treatment.1 This analysis supports the use of leriglitazone to treat pediatric patients with progressive cALD, a rapidly fatal neurodegenerative phenotype of X-linked Adrenoleukodystrophy characterized by inflammatory brain demyelination.2

Among the 11 evaluable patients that demonstrated lesion growth deceleration or disease arrest (95% confidence interval [CI]: 71.5, 100), 5 showed arrested disease (45.5%, 95% CI, 13.9–68.4%) and all had met the pre-defined continuation criteria for the trial. Notably, the median change from baseline was 0.0 (0.0–1.0) for the neurological function score (NFS) and 0.0 (0.0–3.0) for the Loes score (LS).

The Department of Pediatrics has launched a new pilot post-baccalaureate program for individuals from backgrounds underrepresented in medicine and science, socioeconomically disadvantaged backgrounds, and persons with disabilities who intend to pursue medical school training. The program is designed to diversify the medical and scientific workforce and will offer non-academic professional development to selected individuals currently employed within the Department of Pediatrics. Eligible candidates must be currently employed as a research assistant, research technician, or simulation technologist and must have graduated from an undergraduate institution with a minimum GPA of 3.2, with intentions of pursuing a MD or MD/PhD degrees. The program will provide selected candidates with resources for the Medical College Admissions Test, individualized and group mentorship/coaching meetings, and information on interview skills/strategies, time and stress management, financial aid, and critical analysis and reasoning.

Dr. Stefan Worgall, Distinguished Professor of Pediatric Pulmonology and Division Chief of the Pediatric Pulmonology, Allergy and Immunology at Weill Cornell Medicine and has been awarded an R01 by the National Institute of Health for his study, “Respiratory sphingolipid synthesis involved in airway hyperreactivity and viral-triggered asthma.”

For years, researchers have studied the genetic factors that contribute to childhood asthma, a disease that affects millions of children worldwide. Through their research, they have discovered that changes in sphingolipid production may play a key role in the development of asthma. The researchers have found that children with asthma have decreased sphingolipid synthesis, especially in the presence of common asthma risk genotypes. They have also found that rhinovirus , the most common trigger for asthma attacks in children, may further impair sphingolipid synthesis.

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