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相关概念视频

Prescription, Nonprescription and Orphan Drugs01:02

Prescription, Nonprescription and Orphan Drugs

714
Prescription drugs require a prescription from a medical practitioner and can only be obtained from a pharmacy. They have many applications, including treating pain, anxiety, and hypertension.
The misuse and addiction to prescription drugs is a growing problem that can affect people of all age groups, specifically teenagers. This can happen when prescription medications are used in ways not intended by the prescriber, such as taking someone else's prescription or using medication for...
714
Pharmacovigilance01:19

Pharmacovigilance

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Post-marketing surveillance is a critical component of pharmaceutical regulation, often uncovering unanticipated adverse drug reactions (ADRs) once a drug is widely used over an extended period.
This process, termed pharmacovigilance, aims to detect, evaluate, and minimize harmful effects related to medication use. The data collection for pharmacovigilance depends on spontaneous reporting systems, where healthcare professionals or patients voluntarily report suspected ADRs.
In some cases, there...
815
Drug Regulation01:25

Drug Regulation

1.3K
Drug regulation encompasses the management of drug usage by evaluating its safety and efficacy through assessments conducted by regulatory authorities. Regrettably, the history of drug regulation is marred by several catastrophic events. One such incident is the Elixir Sulfanilamide tragedy, in which the toxic compound diethyl glycol was included in a sweet-tasting medication, leading to numerous fatalities. This event prompted the enactment of the Food, Drug, and Cosmetic Act in 1938. Under...
1.3K
Drug Administration and Therapy Phases: Overview01:26

Drug Administration and Therapy Phases: Overview

446
Drugs, the chemical agents used in diagnosing, treating, or preventing diseases, undergo a four-phase process of development: pharmaceutic, pharmacokinetics, pharmacodynamics, and therapeutic.
The pharmaceutical phase focuses on leveraging the physicochemical properties of the drug to design and manufacture an effective product. Variants include orally administered tablets or capsules, topical creams or ointments, and parenteral-delivery solutions or emulsions.
The pharmacokinetic phase...
446
Prodrugs01:30

Prodrugs

2.6K
Prodrugs are a class of pharmaceutical compounds that undergo a biotransformation process within the body to be converted into a pharmacologically active drug. Prodrugs are designed to improve the therapeutic properties of the parent drug, such as enhancing bioavailability, increasing stability, or reducing toxicity. The concept of prodrugs revolves around modifying the chemical structure of the original drug to make it more effective or convenient for administration.
Prodrugs help overcome...
2.6K
Drug Therapy01:28

Drug Therapy

41
The advent of drug therapy has profoundly shaped modern mental health care, providing targeted treatments for a range of psychological disorders. Psychotherapeutic drugs, classified into antianxiety, antidepressant, and antipsychotic medications, address symptoms across anxiety disorders, mood disorders, and schizophrenia. While these medications have transformed patient outcomes, they require careful management due to their potential side effects and limitations.
Antianxiety Medications
41

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Mavorixafor:首次获得批准

Sheridan M Hoy1

  • 1Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand. dru@adis.com.

Drugs
|July 14, 2024
PubMed
概括

马沃里克萨福 (Mavorixafor) 是一种新的口服疗法,已批准用于WHIM综合征,增加中性粒细胞和淋巴细胞. 这种CXCR4抗剂为患有这种罕见疾病的12岁及以上患者提供了突破性进展.

科学领域:

  • 免疫学 免疫学 免疫学
  • 药理学 药理学是指药理学的学科.

背景情况:

  • WHIM综合征是一种罕见的原发性免疫缺陷,其特征是,低血糖球蛋白血症,感染和骨髓炎.
  • C-X-C 化学因子受体4 (CXCR4) 途径在免疫细胞的贩运和功能中发挥着关键作用.

研究的目的:

  • 总结一下选择性CXCR4抗剂马沃里克萨福的发展情况.
  • 为了突出导致FDA批准mavorixafor治疗WHIM综合征的里程碑.

主要方法:

  • 马沃里克萨福选择性地阻断了C-X-C化学因配体12 (CXCL12) 和CXCR4.4之间的相互作用.
  • 临床开发涉及评估WHIM综合征患者的疗效和安全性.

主要成果:

  • 马沃里克萨福在2024年4月获得FDA批准,作为12岁及以上患者的WHIM综合征的第一个治疗方法.
  • 该疗法可用于增加循环中的成熟中性粒细胞和淋巴细胞.

结论:

  • 马沃里克萨福尔在治疗WHIM综合征方面取得了重大进展.
  • 目前正在进行的临床开发旨在扩大其用于其他慢性中性衰竭疾病的用途.

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