CIENCIASMEDICASNEWS

jueves, 3 de julio de 2025

RARE DISEASE LEARNING CENTERS +++++

RARE DISEASE LEARNING CENTERS Generalized Pustular Psoriasis https://checkrare.com/generalized-pustular-psoriasis/ Generalized pustular psoriasis (GPP) is a rare, severe form of psoriasis characterized by sterile pustules across large areas of skin. Erdheim-Chester Disease https://checkrare.com/erdheim-chester-disease-2/ Erdheim-Chester disease (ECD) is a rare condition affecting many parts of the body. Although diagnosed in some children, it most commonly affects adults. Cutaneous T-Cell Lymphoma (CTCL) Learning Center 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. What Is 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. Recognizing and Diagnosing WHIM Syndrome https://checkrare.com/recognizing-and-diagnosing-whim-syndrome/ The features listed in the acronym (W)arts, (H)ypogammaglobulinemia, (I)nfections, and (M)yelokathexis represent the key manifestations of the disorder. However, not all patients will present with the four key features of WHIM at any given time.

FDA Approves Emapalumab to Treat Still’s Disease +...

https://checkrare.com/ FDA Approves Emapalumab to Treat Still’s Disease By: Madaline Spencer| Published on: Jul 3, 2025

2025 Orphan Drugs: PDUFA Dates and FDA Approvals ++++ ++++

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 Nitisinone for the Treatment of Alkaptonuria https://checkrare.com/fda-approves-nitisinone-for-the-treatment-of-alkaptonuria/ The U.S. FDA has approved Harliku (nitisinone) for the treatment of adult patients with alkaptonuria. FDA Approves Dupixent for the Treatment of Bullous Pemphigoid https://checkrare.com/fda-approves-dupixent-for-the-treatment-of-bullous-pemphigoid/ The U.S. FDA has approved Dupixent (dupilumab) for the treatment of adult patients with bullous pemphigoid. FDA Approves Garadacimab for Hereditary Angioedema https://checkrare.com/fda-approves-garadacimab-for-hereditary-angioedema/ The U.S. FDA has approved Andembry (garadacimab) for the treatment of hereditary angioedema (HAE) in patients ages 12 years and older. CLINICAL PERSPECTIVES Patient Experience With Stevens-Johnson Syndrome https://checkrare.com/patient-experience-with-stevens-johnson-syndrome/ Katie Niemeyer, patient advocate, discusses her experience with Stevens-Johnson syndrome/toxic epidermal necrolysis (SJS/TEN). Evolving Policy Landscapes for Rare Disease Access https://checkrare.com/evolving-policy-landscapes-for-rare-disease-access/ Deb Jennings, Head of North America Patient Services Operations at Kyowa Kirin, discusses evolving policy landscapes for rare disease access. The Role of Mental Health in Rare Disease Patient Outcomes https://checkrare.com/the-role-of-mental-health-in-rare-disease-patient-outcomes/ Sumira Riaz, PhD, Health Psychologist & Patient Engagement Consultant at Unboxed Psychology, discusses the role of mental health in rare disease patient outcomes. The Undiagnosed Disease Network https://checkrare.com/the-undiagnosed-disease-network/ Kimberly LeBlanc, Genetic Counselor, Director of the Undiagnosed Diseases Network (UDN) Coordinating Center at Harvard Medical School, discusses approaching variants of uncertain significance in rare diseases.

Comparative Effectiveness of Medical Therapies for Locally Advanced Gastric Adenocarcinoma

Comparative Effectiveness of Medical Therapies for Locally Advanced Gastric Adenocarcinoma: This systematic review’s (SR) primary goal was to summarize pertinent evidence on the medical treatment for non-metastatic, locally advanced gastric adenocarcinoma to inform interest holders. This review will be used to inform a Clinical Practice Guideline by the American Society of Clinical Oncology (ASCO) on this topic. Specifically, the SR will 1) determine comparative effectiveness and harms of medical therapies and 2) determine how treatment effectiveness and harms vary by tumor-specific variables and subpopulations.

The Performance of Fusion Procedures for Degenerative Disease of the Lumbar Spine

The Performance of Fusion Procedures for Degenerative Disease of the Lumbar Spine: The Spine Patient Outcomes Research Trial (SPORT) established that surgery may have a role in patients with stenosis associated with degenerative spondylolisthesis. However, while the SPORT established a role for surgery in the setting of refractory symptoms of nerve compression, primarily in patients with spinal stenosis, it did not lead to clarity regarding the ideal candidate for decompression alone, decompression with fusion, and the role of instrumentation in fusion procedures. In 2014, reviews conducted for guidelines from the Congress of Neurologic Surgeons and the National Association of Spine Surgeons confirmed that these gaps were still unresolved.