Results 141 to 150 of about 187,417 (303)

Newborn screening for severe combined immunodeficiency in Malaysia: current status, challenges and progress

Frontiers in Immunology
IntroductionEarly diagnosis of Severe Combined Immunodeficiency (SCID) increases survival outcomes and quality of life while significantly minimizing healthcare burden and costs.
Wai Leng Chang, Wai Leng Chang, Lokman Mohd Noh, Amir Hamzah Abdul Latiff, Amir Hamzah Abdul Latiff, Kent Chee Keen Woo, Intan Hakimah Ismail, Intan Juliana Abd Hamid, Sangeetha Siniah, Mohd Azri Zainal Abidin, Marina Sham, Adiratna Mat Ripen, Mohd Farid Baharin, Asrul Abdul Wahab, Zarina Thasneem Zainudeen, Ilie Fadzilah Hashim, Yee Ming Wong, Yee Ming Wong, Mohamad Qazreen Ahmad Shawaludin, Adli Ali, Adli Ali, Adli Ali, Adli Ali   +22 more
doaj   +1 more source

In Utero HSC Transplantation for Sickle Cell Disease: A Potential Therapeutic Approach That Overcomes Complications of Current Therapies

Prenatal Diagnosis, EarlyView.
ABSTRACT Sickle cell disease (SCD) affects millions worldwide but has limited treatment options, most of which carry significant side effects. At present, the only curative treatment for SCD is allogeneic or gene‐modified autologous hematopoietic stem cell (HSC) transplantation (Tx).
Oluwaseun O. Babatunde, Madeleine G. Bibby, Anthony Atala, Graça Almeida‐Porada, Christopher D. Porada   +4 more
wiley   +1 more source

Trajectories of Suicidal Risk Impact Mood Regulation Differently in Patients With a Diagnosis of Bipolar Disorder

Acta Psychiatrica Scandinavica, EarlyView.
ABSTRACT Background Bipolar disorder (BD) carries a suicide risk 20 times higher than the general population, with up to 60% of patients attempting suicide. Current interventions have failed to reduce its incidence; static factors have shown limited predictive utility.
Abigail Ortiz, Ramzi Halabi, Daniel Blumberger, Christina Gonzalez‐Torres, Arend Hintze, Muhammad I. Husain, Mirkamal Tolend, Juveria Zaheer, Martin Alda, Benoit H. Mulsant   +9 more
wiley   +1 more source

Psychedelic‐assisted treatment for substance use disorder: A narrative systematic review

Addiction, EarlyView.
Abstract Background and aims This is the first systematic review of the extant literature on all major psychedelic‐assisted treatment for alcohol use disorder (AUD), tobacco use disorder (TUD) and other substance use disorders (SUD). We aimed to summarise the evidence for efficacy of psychedelic‐assisted treatment for AUD, TUD, and SUD; to evaluate its
Theodore Piper, Francesca Small, Sam Brown, Michael Kelleher, Luke Mitcheson, James Rucker, Allan H. Young, John Marsden   +7 more
wiley   +1 more source

The psychometric properties of validated tools to assess cannabis use disorder: A systematic review and meta‐analysis

Addiction, EarlyView.
Abstract Aims To systematically review the evidence on the psychometric performance and accuracy of screening or diagnostic tools for cannabis use disorder. Method Systematic review and meta‐analysis which included studies conducted in clinical settings, schools, universities, community settings and population‐based surveys in multiple countries and ...
Janni Leung, Daniel Stjepanovic, Stephanie Fong, Dani Dawson, Giang Vu, Tesfa Yimer, Caitlin McClure‐Thomas, Wayne Denis Hall, Gary Chung Kai Chan   +8 more
wiley   +1 more source

Mesenchymal Stem Cells From a Klinefelter Syndrome Patient: Functional Characterization and Therapeutic Implications

Andrology, EarlyView.
ABSTRACT Background Cell therapy, particularly those utilizing mesenchymal stem/stromal cells (MSCs), is gaining traction as a therapeutic option for regenerative treatment in patients with limited therapeutic options. Although the safety of MSC‐based interventions is well established, uncertainties remain regarding how genetic abnormalities and ...
Marzena Zychowicz, Wioletta Lech, Anna Figiel‐Dabrowska, Marta Kot, Magdalena Murzyn, Natalia Malawska, Barbara Pienkowska‐Grela, Zuzanna Sandowska‐Markiewicz, Iwona Ziolkowska‐Suchanek, Agnieszka Zimna, Tomasz Oldak, Natalia Rozwadowska, Anna Sarnowska   +12 more
wiley   +1 more source

SCID without typical SCID immunologic abnormalities [PDF]

The Journal of Pediatrics, 2005
openaire   +1 more source

The association between autism and psychosis and the tools used to measure it: An updated systematic review and meta‐analysis

British Journal of Clinical Psychology, EarlyView.
Abstract Objectives Autistic individuals are at increased risk of psychotic experiences and being diagnosed with psychotic disorders. This association may result from methodological issues, including the misinterpretation of psychosis questionnaires by autistic individuals and clinicians' difficulty distinguishing between the conditions.
Michael R. Miles, Dennis Golm, Emma Palmer‐Cooper   +2 more
wiley   +1 more source

Treosulfan–fludarabine conditioning in infants with severe combined immunodeficiencies: Extended study of the UK paediatric treosulfan study

British Journal of Haematology, EarlyView.
Summary Allogeneic haematopoietic stem cell transplantation (HSCT) is a curative therapy for severe combined immunodeficiency (SCID). Conditioning improves donor engraftment and freedom from immunoglobulin replacement (IgR) but increases the risks of acute and late toxicity. Treosulfan, a reduced toxicity alkylating agent, has emerged as an alternative
Su Han Lum, Sinéad Greener, Irum Latif Memon, Persis Amrolia, Zohreh Nademi, Robert Chiesa, Juliana Silva, Helen Young, Stephen Owens, Eleri Williams, Khuen Foong Ng, Terry Flood, Andrew R Gennery, Sophie Hambleton, Kanchan Rao, Mary Slatter   +15 more
wiley   +1 more source

MYC Addiction as a Targetable Vulnerability in Nelarabine‐Resistant T‐Cell Acute Lymphoblastic Leukemia

Cancer Science, EarlyView.
MYC drives nelarabine resistance in T‐ALL via a hyperactive transcriptional program. Pharmacological degradation of BET proteins dismantles this addiction, restoring drug sensitivity and extending survival in vivo. ABSTRACT Acquired resistance represents a fundamental obstacle limiting the long‐term efficacy of cancer chemotherapy.
Jingjing Gao, Chunxu Lin, Suchang Chen, Qi Mo, Chu Bao, Huali Deng, Ziyin Chen, Weichao Hao, Luyong Zhang, Min Wei   +9 more
wiley   +1 more source