Results 181 to 190 of about 16,796 (211)

Pregnancy in Lysinuric Protein Intolerance Complicated by Immune Dysregulation and Severe Thrombocytopenia. [PDF]

JIMD Rep
McCarron EP, Hill M, Lomas A, Elliott V, Walkinshaw E, Bonnett T, Kuet KP, Tattersall R, Mapplebeck C.   +8 more
europepmc   +1 more source

Clinical Experience With Eliglustat in Children With Gaucher Disease Type 1

American Journal of Medical Genetics Part A, EarlyView.
Erika R. Vucko, Allegra Quadri, Rachel E. Hickey, Carly A. Rasmussen, Monika Izdebski, Joel Charrow, Carlos E. Prada   +6 more
wiley   +1 more source

Integrated Clinical and Proteomic Profiling of CD19 Chimeric Antigen Receptor T Cell Therapy in Progressive Systemic Sclerosis

Arthritis &Rheumatology, EarlyView.
Objective To characterize the clinical, immunologic, and proteomic changes associated with CD19 chimeric antigen receptor T cell therapy in patients with progressive systemic sclerosis (SSc). Methods Patients with progressive SSc received CD19 chimeric antigen receptor (CAR)‐T cell therapy and were observed longitudinally for safety, clinical efficacy,
Chenhan Jia, Wanyi Lin, Zhangyi Zhao, Fenglin Wu, Chaoyu Gu, Yuankai Sun, Zhe Ding, Chang Wei, Xinyu Li, Cuiwei Xie, Peng Xia, Zhen Xia, Xuesong Liu, Shaoying Yang, Chunyan Zhang, Hanlin Yin, Liangjing Lu   +16 more
wiley   +1 more source

A rare case of multiple bladder hemangiomas associated with Glanzmann thrombasthenia: Successful control of refractory hematuria by super selective vesical artery embolization. [PDF]

Urol Case Rep
Chaabouni A, Ghozzia M, Elleuch W, Chaker K, Nouira Y, Hadj Slimen M.   +5 more
europepmc   +1 more source

Promises and Pitfalls of Whole Exome Sequencing in Therapy-Resistant Chronic Thrombocytopenia in Childhood: A Case Report. [PDF]

J Pers Med
Györke E, Benyó G, Árvai KB, Bödör C, Kereskai L, Ábrahám H, Réger B, Egyed B, Ottóffy G.   +8 more
europepmc   +1 more source

When it's not Glanzmann thrombasthenia or Bernard-Soulier syndrome: diagnosing other qualitative platelet disorders. [PDF]

Hematology Am Soc Hematol Educ Program
Hayward CPM, Tasneem S.
europepmc   +1 more source

National Bleeding Disorder Foundation Clinical Practice Recommendations for Laboratory Screening of Iron Deficiency With and Without Anemia in the Inherited Bleeding Disorders Population. [PDF]

Haemophilia
Batsuli G, Lewandowska MD, Lim MY, Tickle K, Baldwin MK, Bender R, Davila J, Hayes B, Jacobson-Kelly A, Kouides P, Lambert MP, Nataraj S, Sholzberg M, Sinha R, Al-Samkari H, Dunn AL.   +15 more
europepmc   +1 more source

Investigation for Bleeding Disorders in Suspected Non-Accidental Intracranial Haemorrhage. [PDF]

J Paediatr Child Health
Seeley PE, Monagle P, Garside L, Russell S, Wiggins M, Bhavnani DC, Pratisto N, Tzioumi D.   +7 more
europepmc   +1 more source

Perioperative hemostasis management in patients with von Willebrand disease undergoing cardiac surgery: A scoping review. [PDF]

JTCVS Open
Loskutov O, Maruniak S, Markov Y, Tkachenko D, Loskutov D, Swol J, Todurov B.   +6 more
europepmc   +1 more source