A "productive" meeting with the FDA has given the firm confidence in its regulatory filing and commercialization plans for ST-920.

Sangamo Therapeutics Announces Important Derisking Milestones in Pathway to Anticipated BLA Submission for ST-920 in Fabry Disease ...

法布里病（FD）患者骨密度问题缺乏关注且无诊疗规范。研究人员对 25 例 FD 患者行双能 X 线吸收法（DXA）扫描研究。结果显示患者低骨密度患病率高，Z、T 评分与 Lyso - GL3、BMI、钙水平相关。为 FD 骨病诊疗提供依据。

Report Ocean’s latest analysis delves into the “Vietnam Fabry Disease Treatment Market” Report from 2024 to 2032, covering an array of market facets such as characteristics, size, growth, segmentation ...

Two people in Oregon have died from a rare, progressive brain disorder similar to "mad cow disease" that leads to dementia and is %100 fatal. The Hood River County Health Department confirmed the ...

We expect investors to focus on the sales performance of Amicus Therapeutics’ FOLD lead marketed drug, Galafold (migalastat), which is approved for Fabry disease, when the company reports ...

In plants, the space between cells is a key battleground during infection. To avoid recognition in this space, a strain of the bacterial tomato disease Pseudomonas syringae manipulates plants by ...

The US Food and Drug Administration (FDA) has awarded breakthrough therapy designation to uniQure's AMT-130 for treating Huntington's disease, a rare neurodegenerative condition. This designation for ...

Background: Neuropathic pain is one of the key features of (classical) Fabry disease (FD). No randomized clinical trials comparing effectiveness of different pain management strategies have been ...

This particular candidate is being developed to treat patients with Fabry Disease. A good update was also provided here a few months ago, whereby the independent data monitoring committee (IDMC ...

诊断延误使患者面临误诊、无效治疗等问题，还会加重病情。法布里病（Fabry disease）是一种 X 连锁隐性遗传的罕见病，由编码 α - 半乳糖苷酶 A（α -Gal A）的 GLA 基因突变引起，会导致多器官损伤。AI 技术有助于加速罕见病诊断，预测疾病进展，确定合适治疗 ...