Search research articles

ABOUT JoVE

Overview Leadership Blog JoVE Help Center

AUTHORS

Publishing Process Editorial Board Scope & Policies Peer Review FAQ Submit

LIBRARIANS

Testimonials Subscriptions Access Resources Library Advisory Board FAQ

RESEARCH

JoVE Journal Methods Collections JoVE Encyclopedia of Experiments Archive

EDUCATION

JoVE Core JoVE Business JoVE Science Education JoVE Lab Manual Faculty Resource Center Faculty Site

Terms & Conditions of Use

Related Concept Videos

Therapeutic Index

Therapeutic Index

The therapeutic index of a drug is a key parameter in pharmacology that quantifies the relative safety of a drug by calculating the ratio between the dose that causes toxicity in half the population (50%) to the dose that proves to be effective for half the population (50%). It provides a spectrum of doses for a particular drug ranging from effective to potentially toxic. To illustrate, consider an anticoagulant agent like warfarin. It possesses a narrow window within its therapeutic index to...

Extraction: Advanced Methods

Extraction: Advanced Methods

Metal ions can be separated from one another by complexation with organic ligands–the chelating agent– to form uncharged chelates. Here, the chelating agent must contain hydrophobic groups and behave as a weak acid, losing a proton to bind with the metal. Since most organic ligands used in this process are insoluble or undergo oxidation in the aqueous phase, the chelating agent is initially added to the organic phase and extracted into the aqueous phase. The metal-ligand complex is...

Overview of Advanced Functional Groups

Overview of Advanced Functional Groups

Functional groups are groups of atoms with specific chemical properties that occur within organic molecules and are sometimes denoted as “R”. Functional groups can “functionalize” a compound by enabling it to adopt different physical and chemical properties.
Types of Advanced Functional Groups
The table below summarizes some of the major functional groups in organic chemistry.

Therapeutic Communication

Therapeutic Communication

Communication is a lifelong learning process. Through therapeutic communication, nurses can collect relevant assessment data, provide education and counseling, and interact during nursing interventions. Sending and receiving messages occur through verbal and nonverbal communication techniques and can happen separately or simultaneously.
Verbal communication depends on language or a prescribed way of using words so that people can share information effectively. The critical aspects of verbal...

Microorganisms in Medicine and Therapeutics

Microorganisms in Medicine and Therapeutics

Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.

Cholinergic Antagonists: Therapeutic Uses

Cholinergic Antagonists: Therapeutic Uses

Antimuscarinic drugs have various therapeutic applications by inhibiting parasympathetic stimulation in different systems. Here are the key therapeutic uses of antimuscarinics:
Respiratory Tract: Ipratropium, aclidinium, and tiotropium treat asthma, chronic bronchitis, and chronic obstructive pulmonary disease (COPD). They protect against bronchoconstriction caused by irritants like cigarette smoke, sulfur dioxide, and ozone. They also help reduce nasopharyngeal...

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by

Same author

Editorial: Amyotrophic lateral sclerosis.

Current opinion in neurology·2022

Same author

Association of Serum GFAP with Functional and Neurocognitive Outcome in Sporadic Small Vessel Disease.

Biomedicines·2022

Same author

CSF levels of SNAP-25 are increased early in Creutzfeldt-Jakob and Alzheimer's disease.

Journal of neurology, neurosurgery, and psychiatry·2022

Same author

Glial fibrillary acidic protein in cerebrospinal fluid of patients with spinal muscular atrophy.

Annals of clinical and translational neurology·2022

Same author

Fast versus slow disease progression in amyotrophic lateral sclerosis-clinical and genetic factors at the edges of the survival spectrum.

Neurobiology of aging·2022

Same author

Cognition in the course of ALS-a meta-analysis.

Amyotrophic lateral sclerosis & frontotemporal degeneration·2022

Search research articles

Related Experiment Video

Updated: Jan 20, 2026

Ocular Therapeutic Delivery and Advanced Tissue Retrieval in Adult Rats

Ocular Therapeutic Delivery and Advanced Tissue Retrieval in Adult Rats

Published on: May 23, 2025

Therapeutic advances in SMA.

Albert C Ludolph¹, Claudia D Wurster

¹Department of Neurology, University of Ulm, DZNE, Ulm, Germany.

Current Opinion in Neurology

|August 20, 2019

Summary

Novel antisense oligonucleotide (ASO) therapies significantly alter the course of spinal muscular atrophy (SMA) in children when initiated early. Further research is needed for adolescent and adult SMA treatment efficacy and delivery methods.

More Related Videos

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts

Published on: May 11, 2018

Development of New Therapeutic Applications Using Microfluidics

Development of New Therapeutic Applications Using Microfluidics

Published on: October 1, 2007

Related Experiment Videos

Last Updated: Jan 20, 2026

Ocular Therapeutic Delivery and Advanced Tissue Retrieval in Adult Rats

Ocular Therapeutic Delivery and Advanced Tissue Retrieval in Adult Rats

Published on: May 23, 2025

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts

Published on: May 11, 2018

Development of New Therapeutic Applications Using Microfluidics

Development of New Therapeutic Applications Using Microfluidics

Published on: October 1, 2007

Area of Science:

Neurology
Genetics
Pharmacology

Background:

Spinal muscular atrophy (SMA) is a genetic disorder affecting motor neurons.
Chromosome 5q deletion is the most common cause of SMA.
Novel therapeutic strategies aim to address the underlying genetic defect or enhance gene function.

Purpose of the Study:

To review the latest advancements in therapies for 5q-associated SMA.
To assess the impact of these novel treatments on the disease's natural history.
To discuss current challenges and future directions in SMA therapeutics.

Main Methods:

Review of current literature on SMA therapies.
Analysis of clinical trial data for antisense oligonucleotides (ASOs) and other emerging treatments.
Discussion of gene replacement strategies and small molecule therapies.

Main Results:

Intrathecally delivered ASOs targeting SMN2 show significant efficacy in altering the natural history of SMA in children.
Earlier initiation of ASO treatment correlates with improved outcomes.
Newborn screening for SMA is being considered due to the effectiveness of early intervention.
Challenges remain in drug delivery for adolescent and adult patients, and long-term efficacy requires further study.

Conclusions:

Novel therapies, particularly ASOs, have revolutionized SMA treatment, especially in pediatric populations.
Further research is essential to optimize drug delivery and assess long-term efficacy in all age groups.
The field of SMA therapeutics is rapidly evolving, necessitating ongoing evaluation and adaptation of treatment strategies.