CIENCIASMEDICASNEWS

viernes, 19 de diciembre de 2025

Are blood pressure disparities affecting mortality (Medscape AI) +... +++ ++ +++

Is Dark Skin the Blind Spot of Dermatology? ++++ +++ +++

Phase 3 Data of Vonvendi for the Treatment of Von Willebrand Disease By: Madaline Spencer| Published on: Dec 19, 2025

https://checkrare.com/phase-3-data-of-vonvendi-for-the-treatment-of-von-willebrand-disease/ Latest Summaries https://checkrare.com/

RARE DISEASE LEARNING CENTERS +++++

CME PROGRAMS Immune Thrombotic Thrombocytopenic Purpura (iTTP) Clinical Research Highlights: ISTH 2025 https://checkrare.com/learning/p-isth2025-module3-immune-thrombotic-thrombocytopenic-purpura-ittp-clinical-research-highlights/ Shruti Chaturvedi, MD, provides a summary of clinically relevant data presented at the International Society of Thrombosis and Haemostatis Congress (ISTH 2025) that can enhance the care of patients with iTTP. CREDITS: 0.5 CME | DURATION: 30 minutes EXPIRES: 9/15/2026 | FEE: $0 Immune Thrombocytopenia Clinical Research Highlights: ISTH 2025 https://checkrare.com/learning/p-isth2025-module2-immune-thrombocytopenia-clinical-research-highlights/ Shruti Chaturvedi, MD, provides a summary of clinically relevant data presented at the International Society of Thrombosis and Haemostatis Congress (ISTH 2025) that can enhance the care of patients with ITP. CREDITS: 0.5 CME | DURATION: 30 minutes EXPIRES: 9/15/2026 | FEE: $0 Hemophilia Clinical Research Highlights: ISTH 2025 https://checkrare.com/learning/p-isth2025-module1-hemophilia-clinical-research-highlights/ Steven Pipe, MD, provides a summary of clinically relevant data presented at the International Society of Thrombosis and Haemostatis Congress (ISTH 2025) that can enhance the care of patients with hemophilia. CREDITS: 0.75 CME | DURATION: 45 minutes EXPIRES: 9/15/2026 | FEE: $0 RARE DISEASE LEARNING CENTERS Cutaneous T-Cell Lymphoma (CTCL) https://checkrare.com/cutaneous-t-cell-lymphoma-2/ Cutaneous T-cell lymphoma (CTCL) belongs to the non-Hodgkin lymphoma class of hematologic T-cell lymphoproliferative disorders. WHIM Syndrome https://checkrare.com/whim-syndrome/ WHIM syndrome is a rare, congenital primary immunodeficiency disorder associated with neutropenia that typically presents in childhood or adolescence.

FDA NEWS & CLINICAL PERSPECTIVES ++++ +++++

FDA NEWS 2025 Orphan Drugs: PDUFA Dates and FDA Approvals https://checkrare.com/2025-orphan-drugs-pdufa-dates-and-fda-approvals/ Rare diseases and orphan drugs are at the forefront of novel development and groundbreaking research. Almost half of all novel medications approved by the U.S. Food and Drug Administration (FDA) are orphan drugs. Below is the list of important regulatory dates for all orphan drugs for 2025. FDA Approves Ziftomenib for Relapsed/Refractory NPM1-Mutated Acute Myeloid Leukemia https://checkrare.com/fda-approves-ziftomenib-for-relapsed-refractory-npm1-mutated-acute-myeloid-leukemia/ The U.S. FDA has approved Komzifti (ziftomenib) for the treatment of relapsed or refractory (R/R) acute myeloid leukemia (AML) with susceptible NPM1 mutation. Darzalex Faspro for Patients With High-Risk Smoldering Multiple Myeloma https://checkrare.com/fda-approves-darzalex-faspro-for-patients-with-high-risk-smoldering-multiple-myeloma/ The U.S. FDA has approved Darzalex Faspro (daratumumab and hyaluronidase-fihj) for the treatment of adult patients with high-risk smoldering multiple myeloma (HR-SMM). FDA Approves Revumenib for the Treatment of Relapsed/Refractory Acute Myeloid Leukemia https://checkrare.com/fda-approves-revumenib-for-the-treatment-of-relapsed-refractory-acute-myeloid-leukemia/ The U.S. FDA has approved Revuforj (revumenib) for the treatment of relapsed/refractory acute myeloid leukemia (AML). CLINICAL PERSPECTIVES Long-Term Data From the AQUARIUS Study of Avacopan in Patients With GPA and MPA https://checkrare.com/long-term-data-from-the-aquarius-study-of-avacopan-in-patients-with-gpa-and-mpa/ Naomi Patel, MD, MPH, Rheumatologist at Massachusetts General Hospital, discusses long-term data from the AQUARIUS study of avacopan in patients with granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA). Hemolytic Disease of the Fetus and Newborn: Outcomes of Intrauterine Transfusion and Patient Experiences https://checkrare.com/hemolytic-disease-of-the-fetus-and-newborn-outcomes-of-intrauterine-transfusion-and-patient-experiences/ May Lee Tjoa, PhD, Senior Global Medical Affairs Leader: Nipocalimab and Maternal-Fetal Immunology at Johnson & Johnson, discusses data on hemolytic disease of the fetus and newborn (HDFN) from the 2025 ISUOG World Congress on Ultrasound in Obstetrics and Gynecology. Rilzabrutinib Approval for Adult Patients With Immune Thrombocytopenia https://checkrare.com/rilzabrutinib-approval-for-adult-patients-with-immune-thrombocytopenia/ Amit Mehta, MD, Medical Oncologist, discusses data that led to the approval of Wayrilz (rilzabrutinib) for adult patients with immune thrombocytopenia (ITP). ISUOG World Congress 2025: Hemolytic Disease of the Fetus and Newborn https://checkrare.com/isuog-world-congress-2025-hemolytic-disease-of-the-fetus-and-newborn/ Jannine Williams, Compound Development Team Leader at Johnson & Johnson, discusses key takeaways from studies on hemolytic disease of the fetus and newborn (HDFN) presented at ISUOG World Congress 2025. Plans for Phase 2/3 Clinical Trial of Bexmarilimab Plus Standard of Care in Patients With Myelodysplastic Syndromes https://checkrare.com/plans-for-phase-2-3-clinical-trial-of-bexmarilimab-plus-standard-of-care-in-patients-with-myelodysplastic-syndromes/ Amer Zeidan, MBBS, MHS, Professor of Internal Medicine at Yale School of Medicine and Chief of the Division of Hematologic Malignancies at Yale Cancer Center, discusses plans for a phase 2/3 clinical trial testing bexmarilimab plus standard of care in patients with myelodysplastic syndromes (MDS).

Rare Disease Focus: Hematologic Disorders +++++

FEATURED ARTICLES Survey Finds Wide-Ranging Impact of X-Linked Hypophosphatemia (XLH) on Patient Experiences https://checkrare.com/survey-finds-wide-ranging-impact-of-x-linked-hypophosphatemia-on-patient-experiences/ Al Freedman, PhD, Rare Disease Psychologist and Rare Dad, and Jill H. Simmons, MD, Endocrinologist at Vanderbilt University Medical Center in Nashville, Tennessee highlight the burden of XLH on personal finances and out-of-pocket costs, overall health—physical, mental, and social—and access to expert care. New Staging Tool Available for Healthcare Providers Treating Patients With CTCL https://checkrare.com/new-staging-tool-available-for-healthcare-providers-treating-patients-with-ctcl/ Pamela Blair Allen, MD, MSc, Hematologist, discusses the new CTCL Staging Tool on PROBEinCTCL.com. Chronic Myeloid Leukemia: Overview, Treatment Options, and Advice for Newly Diagnosed Patients https://checkrare.com/chronic-myeloid-leukemia-overview-treatment-options-and-advice/ Jorge Cortes, MD, Director of the Georgia Cancer Center, provides an overview of chronic myeloid leukemia (CML), discusses treatment options, and offers advice to newly diagnosed patients. Cutaneous T-Cell Lymphoma: Natural History and the Need for Better Diagnostic and Treatment Efficacy https://checkrare.com/cutaneous-t-cell-lymphoma-natural-history-and-the-need-for-better-diagnostic-and-treatment-efficacy/ Henry Wong, MD, PhD, Dermatologist at the San Diego Veterans Hospital and the University of California San Diego (UCSD), discusses cutaneous T-cell lymphoma (CTCL). Chronic Myeloid Leukemia: Diagnosis and Treatment https://checkrare.com/chronic-myeloid-leukemia-diagnosis-and-treatment/ Michael Mauro, MD, Director of the Chronic Myeloid Leukemia Program at Memorial Sloan Kettering Cancer Center, discusses the diagnosis and treatment of chronic myeloid leukemia (CML).