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Medicine

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Gene Therapy, myotubular myopathy, Children, Pediatric, ASPIRO, Virus

New Gene Therapy Trial for Severe Neuromuscular Disorder in Children

Ann & Robert H. Lurie Children’s Hospital of Chicago is one of the few centers participating in ASPIRO, an international Phase 1/2 clinical trial of a gene therapy product called AT132 for X-linked myotubular myopathy – a rare disease characterized by severe muscle weakness, breathing difficulty and early death.

Medicine

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Pediatrics, Pediatrician, Pediatric Surgery, pediatric neurological surgery, pediatric orthopedic surgery, pediatric orthopaedic surgery, post-operative fever

Early Postoperative Fever in Pediatric Patients Rarely Associated With an Infectious Source

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Post-operative fevers in children are rarely due to infection, yet they are often subjected to non-targeted testing. This conclusion has been widely recognized in adult patients undergoing surgery, but this is the first large-scale study to verify this finding in children.

Medicine

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Spinal Muscular Atrophy, SMA, Treatment, Phase 3 clinical trial, spinraza, Children, Pediatric, FDA approved

Drug Improves Muscle Function and Survival in Spinal Muscular Atrophy

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More than half of the babies with infantile-onset spinal muscular atrophy (SMA) who were treated with nusinersen (Spinraza) gained motor milestones, compared to none of the babies in the control group. Infants treated with the drug also had 63 percent lower risk of death. These final results from the 13-month, international, randomized, multicenter, sham procedure-controlled, phase 3 trial called ENDEAR were published in the New England Journal of Medicine.

Medicine

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Cancer, Pediatric, Clinical Trial, Gene Therapy, Brain Tumor, Immune System, Adenovirus

A Virus, a Gene and a Pill Used to Harness the Immune System to Fight Brain Tumor in Children

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The first patient in a new Phase 1 gene therapy trial for pediatric brain tumors underwent a leading-edge procedure at Ann & Robert H. Lurie Children’s Hospital of Chicago. During surgery to remove the brain tumor, the patient was injected with an adenovirus, a common cold virus, at the tumor site. The virus was bioengineered not to cause illness but rather deliver a gene that produces human interleukin 12 (hIL-12), a powerful protein to jumpstart the immune system to kill remaining tumor cells. For the next 14 days, the patient is given a pill – veledimex – to activate the gene and control the immune response, so that the inflammation fights the tumor without overwhelming the rest of the body.

Medicine

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Children, Antibiotics, Emergency Department (ED), Racial and Ethnic Differences

White Children More Likely to Get Unnecessary Antibiotics in Pediatric Emergency Departments

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White children with viral diagnoses treated in pediatric emergency departments were up to twice as likely to receive antibiotics compared to minority children, according to a study published in Pediatrics. Although viral respiratory tract infections do not warrant antibiotic treatment, antibiotics were prescribed for these illnesses to 4.3 percent of white, 1.9 percent of black and 2.6 percent of Hispanic children.

Medicine

Science

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Wilms, Wilms Tumor, Genetic Mutation

Comprehensive Genomic Analysis Offers Insights into Causes of Wilms Tumor Development

Mutations involving a large number of genes converge on two pathways during early kidney development that lead to Wilms tumor

Medicine

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PCORI, Pediatric Surgery, Grant Awards, Grants

Barsness Receives Second PCORI Award to Develop Patient and Family Advisory Board to Help Improve Patient Experience

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Katherine Barsness, MD, MS, pediatric surgeon at Ann & Robert H. Lurie Children’s Hospital of Chicago and Associate Professor of Surgery and Medical Education, Northwestern University Feinberg School of Medicine has received a second funding award from the Patient-Centered Outcomes Research Institute (PCORI). The $25,000 award, provided through PCORI’s “Pipeline to Proposal” program, will support a project that brings together patients and clinicians to discuss ways to improve the pediatric surgery patient experience.

Medicine

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Epilepsy, Children, Precision Medicine, Genetic Testing, Seizures, Diagnosis

Genetic Testing Helps Detect Cause of Early Life Epilepsy

A study published in JAMA Pediatrics supports the use of genetic testing, especially with sequencing, as first-line diagnostic method for young children with seizures. Specific genetic factors were found to be the cause of epilepsy in 40 percent of patients evaluated for first presentation with seizures. Genetic testing also yielded a diagnosis in 25 percent of children who had epilepsy with an otherwise unknown cause.

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Children's Health, Pediatrics, American College Of Surgeons, Surgery, quality of surgical care, Pediatric Surgery

Lurie Children’s Recognized as Level 1 Pediatric Surgery Center by American College of Surgeons for Second Time

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For the second year, Ann & Robert H. Lurie Children’s Hospital of Chicago has been named a Level I pediatric surgery center by the American College of Surgeons (ACS). In 2017 Lurie Children’s became the first children’s hospital in Illinois to earn this status and is currently one of only five in the country. The Level I verification is awarded by a multi-organizational taskforce led by the ACS, the body responsible for setting the nation’s standards for quality of surgical care, practice and training.

Medicine

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Child Abuse, Child Maltreatment, Risk Factors

Study Identifies Commonalities in Fatal or Near-Fatal Child Abuse

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Analysis of fatal and near-fatal physical abuse cases of children under 4 years of age revealed that psychosocial risk factors in the home, such as criminal history, were present in all cases. Two-thirds of the cases with prior medical records available (nine children) involved unexplained or atypical bruising – bruises on non-mobile infants, bruises on the ears, buttocks or eyes, and patterned bruises consistent with inflicted injury. All nine of these children suffered subsequent brain injury, resulting in four deaths. Findings were published in Child Abuse & Neglect.







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