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Related Concept Videos

Drug Administration and Therapy Phases: Overview01:26

Drug Administration and Therapy Phases: Overview

562
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...
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Prescription, Nonprescription and Orphan Drugs01:02

Prescription, Nonprescription and Orphan Drugs

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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...
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Clinical Trials: Overview01:11

Clinical Trials: Overview

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Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
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Preclinical Development: Overview01:28

Preclinical Development: Overview

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Preclinical development consists of a series of tests that ensure the safety and efficacy of a new therapeutic compound before it is tested in humans. There are four main phases to this process. First, safety pharmacology tests are conducted to ensure the drug does not produce any acutely harmful effects. These tests examine parameters such as bronchoconstriction, cardiac dysrhythmias, blood pressure changes, and ataxia. Next, preliminary toxicological testing is performed to determine the...
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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...
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Drug Regulation01:25

Drug Regulation

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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...
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Related Experiment Video

Updated: Jul 26, 2025

Radiosynthesis of 1-2-[18F]Fluoroethyl-L-Tryptophan using a One-pot, Two-step Protocol
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Tofersen: First Approval.

Hannah A Blair1

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

Drugs
|June 14, 2023
PubMed
Summary

Tofersen (Qalsody™) is a new antisense oligonucleotide treatment approved for amyotrophic lateral sclerosis (ALS) in adults with SOD1 gene mutations. This marks a significant milestone in ALS therapy development.

Area of Science:

  • Neurology
  • Genetics
  • Pharmacology

Background:

  • Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease.
  • Mutations in the superoxide dismutase 1 (SOD1) gene are linked to a subset of ALS cases.
  • There is a critical need for targeted therapies for genetic forms of ALS.

Purpose of the Study:

  • To summarize the development milestones of tofersen.
  • To highlight the regulatory approval of tofersen for SOD1-ALS.
  • To provide context for this novel ALS treatment.

Main Methods:

  • Review of preclinical and clinical development data for tofersen.
  • Analysis of regulatory submission and approval processes.
  • Summary of key scientific findings supporting tofersen's efficacy.

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Main Results:

  • Tofersen, an antisense oligonucleotide, targets SOD1 mRNA.
  • Biogen's tofersen received US FDA approval on April 25, 2023.
  • This approval is for adult patients with ALS and a SOD1 gene mutation.

Conclusions:

  • Tofersen represents the first approved targeted therapy for SOD1-ALS.
  • The development of tofersen signifies progress in precision medicine for neurodegenerative diseases.
  • This milestone offers new hope for patients with this specific genetic form of ALS.