Design and Rationale of the APPELHUS Phase 3 Open-Label Study of Factor B Inhibitor Iptacopan for Atypical Hemolytic Uremic Syndrome. [PDF]
, 2023 Atypical hemolytic uremic syndrome (aHUS) is a rare, progressive, and life-threatening form of thrombotic microangiopathy (TMA) which is caused by dysregulation of the alternative complement pathway (AP). Complement inhibition is an effective therapeutic
Bagga, A. +8 more
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Complement inhibition in Myasthenia – from basics to RCT data [PDF]
, 2023 Myasthenia gravis (MG) is the prototypic autoimmune neurological disorder causing fatiguable muscle weakness either limited to the ocular muscles or becoming generalised involving the limb and bulbar muscles.
Jacob, Saiju
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Swiss Survey on current practices and opinions on clinical constellations triggering the search for PNH clones. [PDF]
, 2023 UNLABELLED This national survey investigated the current practice in Switzerland by collecting participants' opinions on paroxysmal nocturnal hemoglobinuria (PNH) clone assessment and clinical practice.
Beauverd, Yan +14 more
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Efficacy of innovative therapies in myasthenia gravis: A systematic review, meta-analysis and network meta-analysis [PDF]
, 2023 Background and purpose: Therapy for myasthenia gravis (MG) is undergoing a profound change, with new treatments being tested. These include complement inhibitors and neonatal Fc receptor (FcRn) blockers.
Espinosa, Pablo Ezequiel +4 more
core +1 more source
Canadian Journal of Health Technologies, 2022 CADTH reimbursement reviews are comprehensive assessments of the clinical effectiveness and cost-effectiveness, as well as patient and clinician perspectives, of a drug or drug class. The assessments inform non-binding recommendations that help guide the reimbursement decisions of Canada's federal, provincial, and territorial governments ...
openaire +2 more sources
Complement Mediated Hemolytic Anemias in the COVID-19 Era : Case Series and Review of the Literature [PDF]
, 2021 The complex pathophysiologic interplay between SARS-CoV-2 infection and complement activation is the subject of active investigation. It is clinically mirrored by the occurrence of exacerbations of complement mediated diseases during COVID-19 infection ...
Barcellini W. +4 more
core +1 more source
Hematology Reports, 2023 Paroxysmal nocturnal hemoglobinuria (PNH), a rare acquired hematologic disorder, can be treated with C5 inhibitors (C5i) such as eculizumab or ravulizumab.
Jesse Fishman +4 more
doaj +1 more source
Update on the diagnosis and treatment of neuromyelitis optica spectrum disorders (NMOSD) – revised recommendations of the Neuromyelitis Optica Study Group (NEMOS). Part II: Attack therapy and long-term management [PDF]
, 2023 International audienceThis manuscript presents practical recommendations for managing acute attacks and implementing preventive immunotherapies for neuromyelitis optica spectrum disorders (NMOSD), a rare autoimmune disease that causes severe inflammation
Aktas, Orhan +139 more
core +1 more source
eJHaem, 2022 A 47‐year‐old male with paroxysmal nocturnal haemoglobinuria (PNH) controlled with routine ravulizumab administration suffered a massive haemolytic crisis due to choledocholithiasis.
Mitsuru Moriyama +6 more
doaj +1 more source
Ravulizumab for the treatment of myasthenia gravis
Expert Opinion on Biological Therapy, 2023 Myasthenia gravis (MG) is a neurological B-cell mediated autoimmune disorder affecting the neuromuscular junction. MG therapeutics have always relied on nonselective immunosuppression with oral steroids and non-steroidal immunosuppressants, mainly with good clinical response.
Vanoli Fiammetta, Renato Mantegazza
openaire +3 more sources