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CHOP Hosts Cure Sickle Cell Walk & Family Fun Day Presented by West Pharmaceutical Services, Raising More Than $242,000 for Research and Care
Children’s Hospital of Philadelphia (CHOP) hosted the Cure Sickle Cell Walk & Family Fun Day presented by West Pharmaceutical Services on Saturday, June7. It was a morning of walking, cheering and celebrating sickle cell warriors and their families at Citizens Bank Park. The event, which included a ceremonial walk to honor those with sickle cell disease, along with fun activities for the whole family, raised more than $242,000 for sickle cell disease research and patient care.
Children’s Hospital of Philadelphia Researchers Find Evidence That Tumor Necrosis Factor Alpha Helps Reduce Anemic Inflammation
Researchers at Children’s Hospital of Philadelphia (CHOP) found compelling preclinical evidence that a molecule called tumor necrosis factor alpha (TNFα), typically known for promoting inflammation, also plays a helpful role in recovery from anemia that is caused by ongoing immune disturbances.
Children’s Hospital of Philadelphia Researchers Find Gene Therapy Shows Long-Term Benefits for Hemophilia B Patients
Researchers at Children’s Hospital of Philadelphia (CHOP) announced the results of the longest follow-up study to date for individuals receiving the gene therapy fidanacogene elaparvovec to treat hemophilia B, a rare, X-linked disorder resulting from a deficiency in coagulation factor IX (FIX) activity.
Researchers at Children’s Hospital of Philadelphia Show Gene Therapy Can Improve Quality of Life for Companion Dogs with Hemophilia
In a study published in the journal Molecular Therapy Methods and Clinical Development, researchers at Children’s Hospital of Philadelphia (CHOP), in collaboration with researchers at the School of Veterinary Medicine at the University of Pennsylvania, found that adeno-associated viral (AAV) gene therapy has the potential to be a life-changing treatment for companion dogs –pets – with severe hemophilia, an inherited bleeding disorder caused by deficiency of clotting factor.
Children’s Hospital of Philadelphia Preclinical Study Demonstrates Improved Gene Therapy Approach to Treating Metachromatic Leukodystrophy
In a preclinical study, researchers at Children’s Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with potentially increased effectiveness and safety for the treatment for metachromatic leukodystrophy (MLD), a rare disease in young children characterized by the deficient activity of a critical enzyme. Without effective treatment, MLD leads to rapidly declining neurological skills in young children and is potentially fatal. The promising findings were published in the journal Molecular Therapy Nucleic Acids.
Children’s Hospital of Philadelphia Preclinical Study Unveils Promising New Treatment Approach for Fanconi Anemia Patients
In a preclinical study, researchers at Children’s Hospital of Philadelphia (CHOP) demonstrated a novel treatment strategy for patients with Fanconi anemia (FA), a rare genetic disease resulting in loss of blood-forming stem cells in the bone marrow. The researchers successfully used in situ mRNA delivered directly into the body by lipid nanoparticles (LNPs) to target bone marrow cells and transiently restore stem cell function in patients with FA.
First of Its Kind Gene Therapy Model Offers Hope for X-Linked Sideroblastic Anemia Treatment
Researchers at Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania Perelman School of Medicine pioneered a first of its kind gene therapy model that offers a potential breakthrough in treating X-linked sideroblastic anemia (XLSA), a rare congenital anemia caused by mutations in the ALAS2 gene crucial for the synthesis of heme, a key compound in hemoglobin. This study marks the first time researchers studied gene therapy to treat this disease, which the authors underscore could have an impact on a broad spectrum of diseases. The research was published today and featured on the cover of the journal Blood.
Beyond Blood Fall 2024 Newsletter
Family Newsletter from the Comprehensive Hemostasis and Thrombosis Center at Children’s Hospital of Philadelphia.
Children’s Hospital of Philadelphia Researchers Find Hemophilia A Drug May Be Effective for Select People with Hemophilia B
Researchers at Children’s Hospital of Philadelphia (CHOP) found that emicizumab, a widely used drug for hemophilia A, could also improve blood clotting for select people with hemophilia B depending on their genes. The preclinical study, recently published in Blood, offers proof that this class of hemophilia A drug could potentially be used to treat hemophilia B.
Children’s Hospital of Philadelphia Researchers Find Cytokine Panels Informed Clinical Management in Nearly 30% of Pediatric Cases
The findings show how more rapid results can improve practice and lead to more accurate and timely diagnoses