Results 191 to 200 of about 267,713 (253)

Efficacy and safety of empagliflozin for treating neutropenia and neutrophil dysfunction in paediatric patients with glycogen storage disease type Ib: A systematic review and meta‐analysis

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
Aims Glycogen storage disease type Ib (GSD‐Ib) is a rare genetic disorder causing neutropenia and neutrophil dysfunction in children. G‐CSF has been the primary treatment, but emerging data support the potential of empagliflozin, an SGLT2 inhibitor, as a promising investigational option.
Elizabeth Iwasyk   +5 more
wiley   +1 more source

Cost‐utility analysis of nusinersen–risdiplam switch in patients with spinal muscular atrophy in Croatia: A discrete event simulation model

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
Introduction In recent years, the treatment of spinal muscular atrophy (SMA), a rare disease, has significantly progressed, improving patients' survival and overall quality of life. However, current SMA treatments are expensive, and some (nusinersen) are very inconvenient for patients.
Andrej Belančić   +4 more
wiley   +1 more source

Zuranolone: A case study in (regulatory) rush to judgement?

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
Abstract Sage, in collaboration with Biogen, submitted a new drug approval for zuranolone for postpartum depression (PPD) and major depressive disorder (MDD) in December 2022. In August 2023, the US Food and Drug Administration granted approval for PPD but denied approval for MDD.
Lisa Cosgrove   +4 more
wiley   +1 more source

Barriers and Facilitators to Surgical Trainee Psychological Safety.

open access: yesJAMA Netw Open
Chen JH   +16 more
europepmc   +1 more source

Efficacy, safety and cost‐effectiveness of CAR‐T therapy

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
CAR T‐cells demonstrate high efficacy in blood cancers, including ALL, MM and DLBCL. Innovations target solid tumours despite challenges such as antigen escape. Combination therapies enhance the delivery and infiltration of CAR T cells. Toxicity, cost and resistance remain major barriers to clinical use.
Emina Karahmet Sher   +7 more
wiley   +1 more source

Exploring new avenues: Psychedelic‐assisted therapy for young people

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
Rates of mental illness in young people are increasing, whereas the development of novel mental health treatments has not significantly progressed. Psychedelic‐assisted therapy, using substances such as psilocybin and 3,4‐methylenedioxymethamphetamine (MDMA), has shown potential in the treatment of mental illnesses in the adult population, including ...
Ioanna Artemis Vamvakopoulou   +3 more
wiley   +1 more source

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