Mitapivat: A Review in Pyruvate Kinase Deficiency in Adults
AbstractMitapivat (Pyrukynd®), an oral, allosteric activator of pyruvate kinase (PK), is approved in the USA for the treatment of haemolytic anaemia in adults with PK deficiency and in the EU and UK for the treatment of PK deficiency in adults. Mitapivat acts by restoring activity of the red blood cell (RBC) PK enzyme, which is dysfunctional due to genetic mutations in thePKLR gene in patients with PK deficiency. In the double-blind placebo-controlled phase III ACTIVATE trial in adults with PK deficiency who were not regularly RBC transfused, mitapivat was superior to placebo in improving haemoglobin levels. In the single...
Source: Drugs - November 22, 2023 Category: Drugs & Pharmacology Source Type: research
Momelotinib: First Approval
This article summarizes the milestones in the development of momelotinib leading to this first approval for MF. (Source: Drugs)
Source: Drugs - November 21, 2023 Category: Drugs & Pharmacology Source Type: research
Aetiology of Significant Liver Test Abnormalities in a Single-Centre Cohort of People with Cystic Fibrosis Exposed to Elexacaftor/Tezacaftor/Ivacaftor, Utilizing the Updated RUCAM
ConclusionsAlternative causes were identified in the majority of patients with clinically significant increases in transaminases following E/T/I, highlighting the importance of thorough investigation. Multidisciplinary assessment involving an experienced hepatologist is crucial in cases of diagnostic uncertainty or suggestion of significant DILI, as discontinuation of therapy can have significant consequences for individuals. (Source: Drugs)
Source: Drugs - November 15, 2023 Category: Drugs & Pharmacology Source Type: research
Sunvozertinib: First Approval
This article summarizes the milestones in the development of sunvozertinib leading to this first approval for metastatic NSCLC withEGFR exon20ins. (Source: Drugs)
Source: Drugs - November 14, 2023 Category: Drugs & Pharmacology Source Type: research
Elranatamab: First Approval
This article summarizes the milestones in the development of elranatamab leading to this first approval for the treatm ent of RRMM. (Source: Drugs)
Source: Drugs - November 4, 2023 Category: Drugs & Pharmacology Source Type: research
Current and Emerging Treatment Options for Uterine Fibroids
AbstractUterine fibroids are the most common benign neoplasm of the female reproductive tract in reproductive age women. Their prevalence is age dependent and can be detected in up to 80% of women by the age of 50 years. Patients affected by uterine fibroids may experience a significant physical, emotional, social, and financial toll as well as losses in their quality of life. Unfortunately, curative hysterectomy abolishes future pregnancy potential, while uterine-sparing surgical and radiologic alternatives are variously associated with reduced long-term reproductive function and/or high tumor recurrence rates. Recently, ...
Source: Drugs - November 3, 2023 Category: Drugs & Pharmacology Source Type: research
Correction to: Lotilaner Ophthalmic Solution 0.25%: First Approval
(Source: Drugs)
Source: Drugs - October 31, 2023 Category: Drugs & Pharmacology Source Type: research
