Latest News
Muscle MRI May Be a Useful Biomarker for LOPD Diagnosis in Children
Proposed Spectrometry Method May Detect PD Biomarkers
Electronic PRO System Shows High Feasibility and Performance in Patients With PNH
Rare Case of NMOSD With Peripheral Nerve Damage Reported
Interim PET/CT Parameters Could Help With DLBCL Prognosis
Photodynamic Therapy Plus Chemotherapy May Improve Survival in ECC
Study Sheds Light on How Tumor Cells Escape the Immune System in DLBCL
What to Consider When Selecting a Drainage Technique for CCA
Is the Current Node Staging System for CCA Enough?
New Research Demonstrates the Role of Immunoglobulin M in Autoimmune Disease
Caregivers of People With hATTR Report Anxiety, Stress, and Sleep Problems
Potential Link Between Genotype and Phenotype in TFP Deficiency Identified
Concomitant Eculizumab and IST May Be Safe and Effective in Patients With PNH
Healthy Pregnancy Outcomes Possible in Patients With Autoimmune Hemolytic Anemia
Proximal Complement Inhibitors Could Address Limitations in PNH Treatment
MG Treatment Schemes in the US May Have Gaps
MG-Medication Interaction Tool Reduces Prescription of High-Risk Medication
Disease Severity Determines Quality of Life in MG
Study Supports the Use of Efanesoctocog Alfa for Bleeding Prevention in Hemophilia
Latest Features
Latest Patient Perspectives
Knowing the Difference Between CAD Symptoms and a Heart Attack
Living With hATTR Can Be a Financial Nightmare
The Importance of Self-Care While Living With Pompe
What Cleaning House Is Like for Me as an AATD Patient
Rare Care Podcast
Browse Rare Disease Advisor’s engaging “Rare Care” podcast series, featuring interviews with leading researchers, stakeholders, and influencers within the rare disease community.
Latest HCP Insights
Is Gut Microbiota Involved in Pulmonary Arterial Hypertension?
Pulmonary arterial hypertension (PAH) is primarily associated with abnormal changes to the pulmonary vasculature, which eventually leads to irreversible right heart failure. Pathologists characterize endothelial dysfunction and pulmonary arterial fibrosis as the main drivers of vascular stiffening. Over the last few years, scientists have painstakingly put together a picture of the network of pathological processes that…
The Dangers of Delayed Diagnosis in Acquired Hemophilia A
Acquired hemophilia A differs from classical hemophilia A in that it occurs in individuals without a previous history of bleeding. Both diseases involve neutralizing autoantibodies acting against coagulation factor VIII (FVIII). However, in acquired hemophilia A, patients typically present with an isolated prolonged activated partial thromboplastin time (aPTT) that is later discovered to be due…
Existing Immunosuppressive Strategies for Treating Myasthenia Gravis
Myasthenia gravis is a neuromuscular disorder driven by autoimmunity against the postsynaptic neuromuscular junction of skeletal muscles. The clinical manifestations of myasthenia gravis are broad, but generally it can be characterized as being fluctuating, chronic, and oftentimes carrying a poor prognosis. “[Myasthenia gravis] impairs [neuromuscular junction] transmission and can manifest in a spectrum ranging from…
Treating Advanced Medullary Thyroid Carcinoma
The history of medicine, stretching as far back as when our human ancestors first roamed the earth, is a poignant volume of how far we have come, and how far we have yet to go. Take cancer, for example. In the past, when life expectancy was drastically lower, cancer was seen as less of an…
The Evolving Face of Cystic Fibrosis Care
I distinctly remember whispers when I was in medical school about potentially groundbreaking treatments in the works that might finally make cystic fibrosis an illness of the past. Regrettably, these predictions have not manifested; cystic fibrosis is still very much a condition that carries a high morbidity and mortality rate. It is tempting to desire…
Targeting DNA Damage Response as a Therapeutic Strategy in CCA
Cholangiocarcinoma (CCA) is an aggressive type of cancer with limited therapeutic options available. When possible, patients are offered surgical resection, whereas unresectable cases are managed with adjuvant treatment. Chemotherapy or radiotherapy are usually used to increase the chances of recovery and improve patient's quality of life and survival. However, despite the recent advances, the survival…