CHILDREN'S HOSPITAL OF PHILADELPHIA

🇺🇸United States
Ownership
-
Established
1855-01-01
Employees
-
Market Cap
-
Website
http://www.chop.edu/
forbes.com
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Five Of The Most Important Medical Breakthroughs Of 2024

Recent medical breakthroughs include CRISPR therapy for sickle cell anemia, stem cell treatment restoring vision, artificial ovaries for fertility, CAR T-cell therapy for brain cancer, and gene therapy enabling a deaf child to hear.
rnz.co.nz
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Kennedy's lawyer has asked the FDA to revoke its approval of the polio vaccine

Trump praises polio vaccine as 'greatest thing,' while lawyer Aaron Siri petitions FDA to revoke US vaccine approval. Siri, affiliated with Trump's HHS pick Robert F. Kennedy Jr., seeks withdrawal of inactivated polio vaccine approval until safety trials are conducted. Kennedy, a vaccine skeptic, supports informed choice. Polio vaccination is hailed as a global public health achievement, though Siri's petition highlights lack of placebo-controlled trials, which experts deem unethical. The inactivated polio vaccine used in the US is safer than the oral version, though it doesn't stop virus transmission.
cancer.gov
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Helping Kids with Cancer Speak for the Supportive Care They Need

Two studies found that using SSPedi, a tool for young cancer patients to report symptoms, led to fewer symptoms and better supportive care. The trials, published in JAMA and JAMA Pediatrics, showed that daily symptom reporting reduced distress and improved symptom management. The findings suggest that patient-reported outcomes can significantly improve care for young cancer patients.
news-medical.net
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New protein platform enhances cancer immunotherapy

Researchers from CHOP and Stanford revealed TRACeR-I's molecular structure, a protein platform for reprogramming immune responses, which could optimize cancer treatment designs. TRACeRs act as 'master keys' for MHC proteins, overcoming historical hurdles in MHC-targeting molecules, with high specificity and broad compatibility.
themunicheye.com
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Innovative Precision Medicine Achieves Promising Outcomes for Infantile Fibrosarcoma and ...

Larotrectinib, an oral medication inhibiting cancer cell proliferation, shows significant efficacy in young patients with infantile fibrosarcoma (IFS) and other solid tumors with NTRK gene fusion, with an objective response rate of 94% for IFS and 60% for other tumors. This precision medicine approach may revolutionize treatment protocols, potentially minimizing chemotherapy needs in pediatric patients.
statnews.com
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Johnson & Johnson seeks first drug approval for treatment of smoldering myeloma

Johnson & Johnson seeks approval for Darzalex to treat high-risk smoldering myeloma, with AQUILA trial showing reduced risk of disease progression. GSK's Blenrep, after withdrawal, shows improved survival in DREAMM-7 trial. Emerging efforts aim to ease conditioning for genetic treatments in sickle cell disease. Kura Oncology's menin inhibitor shows promising results in AML, facing competition from J&J and Syndax. Pfizer's Oxbryta withdrawal for sickle cell disease remains unexplained.
prnewswire.com
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Children's Hospital of Philadelphia Researchers Find Precision Medicine Treatment ...

Larotrectinib, an oral drug targeting NTRK gene fusion, was highly effective in pediatric patients with newly diagnosed infantile fibrosarcoma or other solid tumors, potentially replacing chemotherapy. The study, published in the Journal of Clinical Oncology, showed 94% objective response rate in IFS patients and 60% in other solid tumors, with 82.2% and 80% two-year event-free survival respectively.

Survey: titrating trofinetide improves tolerability among Rett syndrome patients

A survey at the AES 2024 Annual Meeting found that 86% of prescribers reported titrating trofinetide improved tolerability in Rett syndrome patients, reducing diarrhea severity and treatment discontinuations, and enhancing quality of life for patients and caregivers.
quantisnow.com
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Long-Term Follow-Up Data Continue to Support Beti-Cel as a Potentially Curative Treatment for Beta-Thalassemia

Beti-cel (ZYNTEGLO™) treatment for beta-thalassemia shows durable transfusion independence and normal hemoglobin levels up to 10 years post-treatment, with a favorable safety profile. 81% of participants have over 5 years of follow-up, and 90.2% achieved transfusion independence. Beti-cel is a one-time gene therapy approved for adult and pediatric patients requiring regular red blood cell transfusions.
newswise.com
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New Research from Children's Hospital of Philadelphia

CHOP and COG Phase 3 study shows blinatumomab plus chemotherapy significantly improves 3-year disease-free survival (DFS) in newly diagnosed NCI standard risk B-cell acute lymphoblastic leukemia (B-ALL) pediatric patients, with DFS rates of 96.0% vs. 87.9% with chemotherapy alone.
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