Tamires Silva Alves,Ana Luíza Fonseca Siqueira,Roberto Giugliani
Tamires Silva Alves
Acid sphingomyelinase deficiency is an ultra-rare disease characterised by generalised storage of sphingomyelin, caused by deficiency of the lysosomal enzyme acid sphingomyelinase, owing to the presence of biallelic pathogenic variants in t...
Lisaftoclax: First Approval [0.03%]
利沙氟星：首次批准
Yahiya Y Syed
Yahiya Y Syed
Lisaftoclax is a B-cell lymphoma 2 (BCL-2) inhibitor developed by Ascentage Pharma for the treatment of haematological malignancies. It is administered using a 5-day ramp-up schedule, reaching the target dose on day 6. Lisaftoclax received ...
Joseph J Tonge,Irina Bacila,Nils P Krone
Joseph J Tonge
Congenital adrenal hyperplasia (CAH) is a group of autosomal recessive endocrine disorders caused by impaired steroidogenesis and insufficient cortisol production from the adrenal cortex. The most common form of CAH is 21-hydroxylase defici...
Stephanie Young Moss,Angie Lee,James A Simon
Stephanie Young Moss
Vasomotor symptoms (VMS) are considered the cardinal symptoms of menopause, affecting up to 80% of American women at some point during the menopausal transition. VMS, particularly if they are moderate to severe, frequent, or cause sleep dis...
Matt Shirley
Matt Shirley
Mazdutide (Xinermei®) is a dual glucagon receptor (GcgR) and glucagon-like peptide-1 receptor (GLP-1R) agonist being developed by Eli Lilly and Company along with Innovent Biologics for use in weight management in adults with obesity or ov...
Simon Fung
Simon Fung
Ifupinostat (; Betlin) is a first-in-class dual phosphoinositide 3-kinase α (PI3Kα)/histone deacetylase (HDAC) inhibitor being developed by BeBetter Med for the treatment of relapsed or refractory diffuse large B-cell lymphoma. On the 30t...
Preethy E Abraham,Douglas B Johnson
Preethy E Abraham
Immune checkpoint inhibitors (ICIs) produce often durable responses in many cancer types but are associated with autoimmune-like toxicities. These immune-related adverse events (irAEs) occur in multiple organ systems and often improve with ...
Clinical Efficacy, Safety and Imaging Effects of Oral Valiltramiprosate in APOEε4/ε4 Homozygotes with Early Alzheimer's Disease: Results of the Phase III, Randomized, Double-Blind, Placebo-Controlled, 78-Week APOLLOE4 Trial [0.03%]
阿普利姆皮格特口服治疗早老素ε4/ε4纯合子型老年痴呆症患者的临床疗效、安全性及影像学效应：APOLLOE4为期78周的III期随机双盲安慰剂对照试验结果
Susan Abushakra,Aidan Power,David Watson et al.
Susan Abushakra et al.
Background: The apolipoprotein E ε4 (APOE ε4) allele is the strongest genetic risk factor for Alzheimer's disease (AD), with homozygotes accumulating a high burden of cerebral beta-amyloid (Aβ) pathology. Valiltramipro...
Hannah A Blair
Hannah A Blair
Sebetralstat (EKTERLY®), an orally available plasma kallikrein inhibitor, is being developed by KalVista Pharmaceuticals for the on-demand treatment of acute attacks of hereditary angioedema (HAE). On 7 July 2025, sebetralstat received its...
Onradivir: First Approval [0.03%]
恩曲他滨：首次批准
Arnold Lee
Arnold Lee
Onradivir (®, Anruiwei) is a small molecule RNA polymerase inhibitor that potently binds to the PB2 cap-binding domain of RNA polymerase to inhibit the replication of the influenza A virus. It was developed by Guangdong Raynovent Biotech a...