Current treatment options for treating OPA1-mutant dominant optic atrophy [0.03%]
OPA1突变导致的显性视神经萎缩的现有治疗选择
Lorenzo Ferro Desideri,Carlo Enrico Traverso,Michele Iester
Lorenzo Ferro Desideri
Dominant optic atrophy (DOA) is caused by OPA1 gene mutation, and it represents one of the most frequently diagnosed forms of hereditary optic neuropathies. This neurodegenerative disorder typically occurs in the first decades of life, and ...
M W McCarthy
M W McCarthy
On December 22, 2021, the United States Food and Drug Administration (FDA) issued an emergency use authorization (EUA) for nirmatrelvir/ritonavir (Paxlovid) for the treatment of mild to moderate coronavirus disease 2019 (COVID-19). The drug...
Renan Öziskender,Ahmet Emre Eşkazan
Renan Öziskender
The introduction of tyrosine kinase inhibitors (TKIs) represents a new era in the management of chronic myeloid leukemia (CML). Despite their long clinical success, point mutations emerging before or during TKI treatment remain an obstacle ...
Shengwu Liu,Kristen E Lowder
Shengwu Liu
Tyrosine kinase inhibitors (TKIs) have provided great benefit for patients with EGFR-mutant non-small cell lung cancer (NSCLC). While prior TKIs have demonstrated limited efficacy against exon 20 insertion mutations of EGFR (EGFR Ex20Ins), ...
Somapacitan: a long-acting growth hormone derivative for treatment of growth hormone deficiency [0.03%]
索马帕西坦：一种长效生长激素衍生物用于治疗生长激素缺乏症
David M Paton
David M Paton
Growth hormone deficiency (GHD) is characterized by inadequate HG production from the anterior pituitary gland. Adult patients with GHD have increased fat mass, an abnormal lipid profile, decreased lean body mass and bone mineral density, d...
Disitamab vedotin, a novel HER2-directed antibody-drug conjugate in gastric cancer and other solid tumors [0.03%]
新型HER2导向抗体药物偶联物恩赫塞まさぶ杜匹坦恩治疗胃癌及其他实体肿瘤的研究进展
Yixuan Hu,Yinxing Zhu,Xiaowei Wei et al.
Yixuan Hu et al.
Antibody-drug conjugates (ADC), a combination of cytotoxic drugs and antibodies, have emerged as a rising star in cancer therapy. Disitamab vedotin (RC48), a novel ADC targeting human epidermal growth factor receptor 2 (HER2), is currently ...
Giovanni Manfredi Assanto,Emilia Scalzulli,Massimo Breccia
Giovanni Manfredi Assanto
Despite the fact that, in the last years, life expectancy of chronic myeloid leukemia (CML) patients has reached that of the normal population, a significant proportion of CML patients is likely to fail treatment with first- or second-gener...
Matteo Tagliapietra
Matteo Tagliapietra
Alzheimer's disease (AD) is the most frequent neurodegenerative condition, the most common cause of dementia, and a leading cause of disability and death globally. Mounting evidence supported accumulation of amyloid β (Aβ) as the primary ...
Medicines for Europe - 16th Legal Affairs Conference (June 29-July 1, 2022 - Barcelona, Spain) [0.03%]
欧洲药品法制会议第十六次会议(2022年6月29日至7月1日，西班牙巴塞罗那)
Monia Tumminello
Monia Tumminello
Medicines for Europe held its 2022 annual conference in Sitges, Spain, from June 29 to July 1. Many topics were discussed including future-proofing healthcare systems in the E.U., methods to build a sustainable European ecosystem to incenti...
David M Paton
David M Paton
Achondroplasia is the commonest form of dwarfism and results from a mutation in the fibroblast growth factor receptor 3 (FGFR3) gene on chromosome 4p16.3. The mutation is at nucleotide 1138 resulting in a G-to-A transition (134934.0001). Th...