The Rare Disease Report page is a resource for medical news and expert insights on rare diseases. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for sickle cell disease, multiple myeloma, and more.
July 5th 2023
Dr. Juliana Biondo discusses what the future of treatment for hemophilia A looks like based on ongoing trials.
Taking Pompe Disease Management to the Next Level: Optimizing Clinical Assessments and Treatment Decision-Making
C.R.U. Eye Symposium (Current, Relevant, Useful)
Patient, Provider, and Caregiver Connection™: Incorporating the Patient Journey into HS Diagnosis and Management Practices
Advances In™ Desmoid Tumors: Recognizing Disease Burden, Pathophysiology, and Targeted Treatments in Development
BURST CME™: Taking ALS Management to the Next Level
Improving Outcomes in Autoimmune Hemolytic Anemias at the Intersection Between Hematology and Oncology Care
Among other associations, those in educational attainment, employment status, mental health, and pain episode frequency and severity highlight the need for comprehensive care that considers the psychological well-being of individuals with SCD.
Gain-of-function STAT4 variants in patients with disabling pansclerotic morphea suggest a potential therapeutic role for JAK inhibition, resulting in improved clinical symptoms and inflammatory markers.
Celularity's clinical trial of CYNK-001 for relapsed and refractory AML showed excellent tolerability, significant graft-versus-leukemia effects, and potential for flexible therapeutic development.
The phase 3 clinical trials evaluating ziritaxestat for treating idiopathic pulmonary fibrosis showed no improvement in clinical outcomes leading to the termination of the trials due to lack of efficacy.
An analysis of data from the National Inpatient Sample offers an overview of risk for adverse delivery outcomes associated with Takayasu arteritis among a real-world cohort of US-based patients.
Patients with myasthenia gravis have a higher risk of COVID-19-associated emergency department visits, hospital admissions, and 30-day mortality, but vaccination acceptance rate was high among the population
Results show a high level of adherence to hydroxyurea medication is essential for lowering the incidence of negative clinical outcomes like VOCs and acute chest syndrome in children with sickle cell anemia.
Investigators address 2 questions: How can we better understand the biology of these rare disorders? How can we stimulate research and optimize the regulatory process to improve diagnosis and targeted treatment?
Positive phase 2 data from 2 studies on the investigational monoclonal antibody felzartamab designed to deplete CD38+ plasma cells, demonstrated reduced pathogenic antibody levels in primary membranous nephropathy.
A silent disease characterized as a chronic cutaneous neuropathy, notalgia paresthetica has an unmet need yet to be fully understood and, dermatologists argue, an FDA-approved treatment could help improve understanding of the etiology and prevalence of this disease.
The treatment is a first-in-class peptide derived from mTB Chaperonin 60.1 that is often involved in resetting the immune system. The treatment is being evaluated in patients with eosinophilic esophagitis and allergen sensitivity.