Results 61 to 70 of about 32,062 (289)

Intracranial haemorrhage in a neonate with severe haemophilia A: A case report and literature review

Zdravniški Vestnik, 2012
Background: Intracranial haemorrhage (ICH) is a serious complication of haemophilia that affects 3.5–4 % of all boys with haemophilia during the neonatal period.
Barbara Faganel Kotnik, Lidija Kitanovski, Vladan Rajić, Darja Paro Panjan, Janez Jazbec, Majda Benedik Dolničar   +5 more
doaj  

Joint protection in haemophilia

, 2011
Haemarthroses (intra-articular haemorrhages) are a frequent finding typically observed in patients with haemophilia. Diagnosis and treatment of these bleeding episodes must be delivered as early as possible.
Valentino, L. A., U. HEDNER, K. KNOBE, V. JIMENEZ-YUSTE, J. A. AZNAR, Hedner, Ulla,, Hedner, Ulla, Knobe, Karin, Ljung, Rolf,, Rodriguez-Merchan, E. C., A. CAFFARINI, F. QUEROL, Aznar, J. A., Caffarini, A., C. A. LEE, R. LJUNG, E. SANTAGOSTINO, Jimenez-Yuste, V., Ljung, Rolf, Santagostino, E., Lee, C. A., E. C. RODRIGUEZ-MERCHAN, L. A. VALENTINO, Querol, F., Lund University.   +24 more
core   +1 more source

Prime editing in neuropsychiatric disorders: From mutation‐specific target selection to clinical translation

Neuroprotection, EarlyView.
Abstract Prime editing, a novel clustered regularly interspaced short palindromic repeats (CRISPR)‐based technology, fuses a reverse transcriptase (RT) to an engineered CRISPR‐associated protein 9 (Cas9) and uses a prime editing guide RNA (pegRNA)‐encoded template.
Tianshan Ji, Yuan Zhang, Jinyi Zhao, Yiping Lu, Chengkun Wang   +4 more
wiley   +1 more source

Clinical, humanistic, and economic burden of severe haemophilia B in adults receiving factor IX prophylaxis: findings from the CHESS II real-world burden of illness study in Europe

Orphanet Journal of Rare Diseases, 2021
Background Real-world studies of the burden of severe haemophilia B in the context of recent therapeutic advances such as extended half-life (EHL) factor IX (FIX) products are limited.
Tom Burke, Sohaib Asghar, Jamie O’Hara, Margaret Chuang, Eileen K. Sawyer, Nanxin Li   +5 more
doaj   +1 more source

Hemophilia A: An Ideal Disease for Prenatal Therapy

Prenatal Diagnosis, EarlyView.
ABSTRACT Hemophilia A (HA) is the most common inherited coagulation defect. Current state‐of‐the‐art treatment consists of frequent administration of prophylactic infusions of coagulation factor VIII (FVIII) protein or bispecific antibodies that replace the cofactor function of FVIIIa to maintain hemostasis. However, these treatments are far from ideal,
Christopher D. Porada, Anthony Atala, Graça Almeida‐Porada   +2 more
wiley   +1 more source

Child Health Challenges, Ecosystem Mismatches, and the Case for State‐Level Policy Innovation

The Milbank Quarterly, EarlyView.
Policy Points From 2011‐2023, US children's health deteriorated across >170 indicators. Chronic conditions rose from 25.8% to 31%, risk of anxiety tripled, and autism and learning disability more than doubled. Policies based on biomedical models have failed to address these trends.
NEAL HALFON, SHIRLEY RUSS
wiley   +1 more source

A Rare Case of Acquired Haemophilia A Following Sigmoid Adenocarcinoma: Case Report and Literature Review

Asia-Pacific Journal of Clinical Oncology, EarlyView.
Acquired haemophilia A (AHA) is a rare complication of malignancy. We report a case of AHA secondary to colorectal cancer which presented as post‐operative bleeding, progressive activated partial thromboplastin time prolongation, precipitous factor VIII (FVIII) decline and inhibitor development.
Benjamin Reardon, Harini Haran, Jennifer Curnow   +2 more
wiley   +1 more source

Severe haemophilia A in a preterm girl with Turner syndrome: case report – a diagnostic and therapeutic challenge for a paediatrician (Part 2)

Italian Journal of Pediatrics, 2021
Background Haemophilia A is an X-linked genetic condition which manifests itself mainly in male children in the first 2 years of life, during gross motor skill development. This disorder is rare in females.
Berendt Agnieszka, Wójtowicz-Marzec Monika, Wysokińska Barbara, Kwaśniewska Anna   +3 more
doaj   +1 more source

Management of carriers and babies with haemophilia [Elektronisk resurs]

, 2008
Although up to 30% of babies born with haemophilia do not have a family history of the disorder, the remaining 70% are born in families where haemophilia has been diagnosed.
Lavery, S A, Ljung, Rolf,, Street, A M, Lund University.   +3 more
core   +1 more source

Red lines and green lights: Gene therapy for inherited erythroid disorders beyond the haemoglobinopathies

British Journal of Haematology, EarlyView.
Gene therapy is revolutionizing treatment paradigms for haemoglobinopathies, establishing a translational framework for disorders that impact red blood cell development. In their paper, Joshi et al. describe the preclinical and early clinical landscape of gene therapies for non‐haemoglobinopathy erythroid disorders and highlight common thematic ...
Gaurav Joshi, Nandakumar Natarajan, Kathryn E. Dickerson, Richard A. Voit   +3 more
wiley   +1 more source