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Our Latest President's Message
From Compassion to Discovery: The Invaluable Impact of Our Clinical Research Coordinators
Science Showcase
- Follow Along as Our Researchers Present at PAS 2024
News from the Manne Research Institute Pillars
- Novel Biological Mechanism Discovered That Could Lead to New Treatments for Neurological Disorders, Cancers
- Lurie Children’s First in Illinois to Treat Duchenne Muscular Dystrophy with FDA-Approved Gene Therapy
- Mental Health Emergencies in Kids Were More Severe During the Pandemic
- Gotta Go? New Bladder Device Lets You Know
Manne Research Institute in the Media
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From Compassion to Discovery: The Invaluable Impact of Our Clinical Research Coordinators
Manne Research Institute's latest President's Message from Dr. Patrick Seed
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Enhancing patient and family understanding of clinical trials is crucial for broadening participation and the number of studies conducted at Stanley Manne Children's Research Institute. Our talented clinical research coordinators create and nurture vital collaborations between the families and our research teams and are integral to the trial work we do. In the latest Manne Research Institute President’s Message, President and Chief Research Officer Patrick Seed, MD, PhD, introduces us to Vanessa Rodriguez and Yolanda Santiago, two of our dedicated clinical research coordinators who bring scientific knowledge, compassion for our patients and families, and a collaborative spirit to raise awareness of how clinical study participation advances our groundbreaking medical research. | |
View previous editions of Dr. Seed's President's Message here. | |
Follow Along as Our Researchers Present at PAS 2024 | | |
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NEWS FROM THE RESEARCH PILLARS | |
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The lab of Yongchao C. Ma, PhD, at Stanley Manne Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital of Chicago discovered a fundamental biological mechanism that could lead to new treatments for neurological diseases, such as spinal muscular atrophy (SMA) and autism, as well as different cancers. The study was published in the journal Human Molecular Genetics.
Dr. Ma’s team found that chemical modification of RNA (called RNA methylation) regulates mitochondrial function. Mitochondria is best known for generating energy in the cell. However, Dr. Ma explains, mitochondria does much more. It also acts as a signaling center that regulates broad biological processes within the cell. Dr. Ma’s lab previously linked mitochondrial dysfunction to the development of SMA and autism, while other labs have implicated it in cancer pathogenesis.
“Our finding establishes a critical link between RNA methylation, mitochondria and diseases that relate to mitochondrial dysfunction, which means that now we have potential for new treatments for many different disorders,” said Dr. Ma, who is the senior author on the study. He holds the Children’s Research Fund Endowed Professorship in Neurobiology at Lurie Children’s and is Associate Professor of Pediatrics, Neurology, and Neuroscience at Northwestern University Feinberg School of Medicine.
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On March 27, 2024, Ann & Robert H. Lurie Children’s Hospital of Chicago treated its first patient with ELEVIDYS (delandistrogene moxeparvovec-rokl), the first gene therapy for Duchenne muscular dystrophy – a rare, genetic disease characterized by progressive muscle damage and weakness. Lurie Children’s is the first in Illinois to administer this treatment after ELEVIDYS received U.S. Food and Drug Administration (FDA) approval in June 2023.
Developed by Sarepta Therapeutics, ELEVIDYS is approved for the treatment of Duchenne muscular dystrophy (DMD) in ambulatory patients aged 4 through 5 years with a confirmed mutation in the DMD gene.
“Our team at Lurie Children’s has had encouraging experience with this gene therapy for Duchenne through our active participation in clinical trials,” said Nancy Kuntz, MD, Director of Muscular Dystrophy Association Care Center at Lurie Children’s and Professor of Pediatrics and Neurology at Northwestern University Feinberg School of Medicine. “Over the past two years, we have treated three boys with DMD with ELEVIDYS as part of a larger clinical trial, and it’s gratifying to see that their muscle strength and function stabilized. Without gene therapy, we would expect to see ongoing deterioration in muscle function in these boys. This therapy is not a cure, and unfortunately it cannot reverse previous muscle damage, but we anticipate that we can slow down the disease enough for science to step in and offer new treatments. This is the beginning of a very exciting journey.”
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A new study found that during the pandemic pediatric emergency departments (EDs) saw more children and adolescents who needed a psychiatric admission, as well as an increase in severe conditions, such as bipolar disorder, schizophrenia and substance use disorders. The higher demand for a psychiatric inpatient bed often exceeded availability, resulting in over 12-hour stays in the ED awaiting admission for nearly 20 percent of children with mental health emergencies in 2022, up from 7 percent before the pandemic. Findings were published in Academic Emergency Medicine.
“Our data shows that pediatric emergency departments saw more severe mental health presentations during the pandemic, even while the actual number of visits decreased in 2022,” said lead author Jennifer Hoffmann, MD, MS, emergency medicine physician at Ann & Robert H. Lurie Children’s Hospital of Chicago and Assistant Professor of Pediatrics at Northwestern University Feinberg School of Medicine. “The dramatic increase in prolonged ED stays attests to the strain on the system and difficulties finding appropriate psychiatric care for children, whether in the hospital or in the community.”
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MANNE RESEARCH INSTITUTE IN THE MEDIA | |
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