Results 121 to 130 of about 20,794 (210)

RNA‐Based Therapies for Inherited Metabolic Disorders

Journal of Inherited Metabolic Disease, Volume 49, Issue 2, March 2026.
ABSTRACT Inherited metabolic disorders (IMDs) are a diverse and complex group of genetic conditions resulting from deficiencies in enzymes, transporters, or cofactors. These deficiencies lead to metabolic dysfunction and severe clinical consequences. Despite significant progress in understanding their molecular basis, treatment options remain limited ...
Reddy Sreekanth Vootukuri, Sonam Gurung, Roopkatha Ghosh, Philippa B. Mills, Julien Baruteau, Haiyan Zhou   +5 more
wiley   +1 more source

Companion Diagnostics in Clinical Therapy: Current Applications and Future Directions

MedComm, Volume 7, Issue 3, March 2026.
Companion diagnostics use biomarker‐based assays to stratify patients for precision therapies, improving outcomes while reducing ineffective treatment and side effects. This review summarizes the evolution, regulatory landscape, clinical applications, and technological platforms of CDx, highlighting their role in biomarker‐driven precision therapy.
Yuesong Wu, Rou Xue, Xiangwen Luo, Jiangnan Liao, Zongbo Zhang, Jinhai Deng, Teng Liu, Xin Li, Zhe‐Sheng Chen, Mingzhu Yin   +9 more
wiley   +1 more source

Concurrent Presentation of von Willebrand Disease and Inflammatory Bowel Disease—A Case Report and Literature Review

eJHaem, Volume 7, Issue 2, April 2026.
Suraj Pratap, Aimee Foord
wiley   +1 more source

Nature Inspired Delivery Vehicles for CRISPR‐Based Genome Editing

Small, Volume 22, Issue 16, 17 March 2026.
The review highlights nature‐inspired nanocarriers for CRISPR delivery, emphasizing viral vectors, extracellular vesicles, liposomes, and lipid nanoparticles. It discusses their roles in improving specificity, minimizing immunogenicity, and overcoming barriers in genome editing. Recent advancements, challenges, and therapeutic applications are explored,
Elizabeth Maria Clarissa, Mamata Karmacharya, Hyunmin Choi, Sumit Kumar, Yoon‐Kyoung Cho   +4 more
wiley   +1 more source

Medical haematology: Repositioning haematology at the centre of medicine

British Journal of Haematology, Volume 208, Issue 3, Page 845-854, March 2026.
The complementary domains of Medical Haematology and Haemato‐Oncology share a biological framework. The left panel depicts red cells, platelets, coagulation, thrombosis and innate immunity, while the right panel shows lymphoid and myeloid malignancies.
Cheng Hock Toh, Imelda Bates, Sue Pavord
wiley   +1 more source

Ageing with Haemophilia: Comorbidities in Focus. [PDF]

Transfus Med Hemother
Rühl H, Marquardt N, Oldenburg J.
europepmc   +1 more source

Haemophilic elbow arthropathy treated with therapeutic arthroscopy - A haemophilia tertiary centre experience. [PDF]

Shoulder Elbow
Hammond RF, Igah I, McLaughlin P, Zu L, Chowdary P, Ahrens P.   +5 more
europepmc   +1 more source

Autoimmune haemophilia.

Blood transfusion = Trasfusione del sangue, 2008
openaire   +2 more sources

Assessment of Static Balance with and Without Cognitive Dual Task in Children with Haemophilia: A Cross-Sectional Study. [PDF]

J Funct Morphol Kinesiol
Chimeno-Hernández A, Alberola-Zorrilla P, Martín Campos J, Carrasco JJ, Pérez-Alenda S, Sánchez Zuriaga D, Aguilar-Rodríguez M.   +6 more
europepmc   +1 more source

Haemophilia. [PDF]

Annals of Internal Medicine, 1981
openaire   +2 more sources