Results 111 to 120 of about 3,536 (218)
Spinal muscular atrophy (SMA) is a severe neuromuscular disease with emerging therapeutic complexity. This review aims to systematically map the global pipeline of investigational treatments for SMA. Using ClinicalTrials.gov and complementary international registries, we identified 21 planned or ongoing interventional trials from 2020 to 2025 targeting
Andrej Belančić +7 more
wiley +1 more source
ABSTRACT Methotrexate (MTX) is a folate antagonist widely used as a disease‐modifying antirheumatic drug (DMARD) in rheumatoid arthritis (RA), as well as in several malignancies and autoimmune disorders. Although low‐dose MTX (5–20 mg/week) is generally considered safe, it has a narrow therapeutic index, and toxicity can occur even at therapeutic doses,
Fatemeh Afra +7 more
wiley +1 more source
ABSTRACT Catastrophic antiphospholipid syndrome (CAPS) is a fulminant and rare variant of antiphospholipid syndrome characterized by rapidly progressive multiorgan thrombosis and a high mortality rate. Its diagnosis is often challenging due to overlapping clinical features with sepsis, thromboembolic disorders, and systemic autoimmune diseases.
Nazmin Ahmed +4 more
wiley +1 more source
Clinical Scoring Systems in Thrombotic Microangiopathies
Thrombotic microangiopathies (TMAs) are a group of rare but potentially lethal hematologic disorders characterized by platelet-rich thrombi in the microvasculature.
Lova Sun +4 more
core +1 more source
ABSTRACT Snakebite envenomation remains a significant yet often overlooked health challenge in Nepal, particularly in its rural and hilly regions. While venom‐induced consumption coagulopathy (VICC) is commonly observed with viper bites, microangiopathic hemolytic anemia (MAHA) is an extremely rare complication, especially with green pit vipers ...
Prabhat Kaphle +10 more
wiley +1 more source
Pregnancy-related thrombotic microangiopathies: Clues from complement biology
International audiencePregnancy is a high-risk period for various types of thrombotic microangiopathies (TMA). The improvement of our understanding of the pathophysiology of TMAs has translated into better management of pregnancy-related TMAs.
Fakhouri, Fadi +2 more
core +1 more source
Shifting From Systemic to Precision‐Targeted Complement Therapies: Opportunities and Hurdles
Complement therapeutics have expanded considerably, but systemic inhibitors remain limited by infection risks, breakthrough events, and loss of physiological functions. Emerging targeted approaches aim for organ‐, tissue‐, or cell‐specific modulation of complement activity, potentially offering greater precision while reducing treatment burden and ...
Marco Mannes +2 more
wiley +1 more source
Etiology and outcomes: Thrombotic microangiopathies in pregnancy. [PDF]
Scully M, Neave L.
europepmc +1 more source
Therapeutic Plasma Exchange in the Elderly: Rare Indications but Good Tolerability
ABSTRACT Therapeutic plasma exchange (TPE) is used for a wide range of indications and is associated with well‐described adverse events. However, no study has specifically focused on tolerance in elderly patients, despite its expected increasing use in this population. We conducted a single‐center retrospective case–control study of all patients aged ≥
Valentin Coirier +11 more
wiley +1 more source

