Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

FDA Approved Drugs: Changes to Approved Drugs01:26

FDA Approved Drugs: Changes to Approved Drugs

293
Post-approval, manufacturers may modify an approved new or generic drug product. Such modifications can encompass alterations in the Active Pharmaceutical Ingredient (API), manufacturing process, formulation, batch size, manufacturing site, and container closure system (FDA Guidance for Industry, April 2004). Often, a drug product may undergo multiple changes.These modifications require careful evaluation to determine their potential impact on the drug product's identity, strength, quality,...
293
Clot Retraction and Fibrinolysis01:16

Clot Retraction and Fibrinolysis

9.7K
After a fibrin clot is formed, the next step is clot retraction, a vital process facilitated by platelet contractile proteins, such as actin and myosin. These proteins pull the fibrin strands closer together and condense the clot. This action reduces the size of the clot, creating a smaller, denser structure that effectively seals off the damaged vessel. Clot retraction consolidates the clot and helps with wound healing by bringing the edges of the damaged blood vessel closer together.
9.7K
Treatment for Pulmonary Arterial Hypertension: Endothelin Receptor Antagonists01:18

Treatment for Pulmonary Arterial Hypertension: Endothelin Receptor Antagonists

511
Endothelins (ETs) are potent vasoactive peptides critical in the human body's various physiological and pathological processes. One of the most promising therapeutic strategies for treating pulmonary arterial hypertension (PAH) involves counteracting the effects of these endothelins using a class of drugs known as endothelin receptor antagonists.
ETs are synthesized through a complex sequence of enzymatic steps, primarily involving an enzyme referred to as endothelin-converting enzyme...
511
Treatment for Pulmonary Arterial Hypertension: Receptor Tyrosine Kinase Inhibitors and Calcium Channel Blockers01:26

Treatment for Pulmonary Arterial Hypertension: Receptor Tyrosine Kinase Inhibitors and Calcium Channel Blockers

597
Receptor tyrosine kinase inhibitors (TKIs) and calcium channel blockers (CCBs) are two critical categories of drugs employed in the treatment of pulmonary artery hypertension (PAH). PAH is a disease that causes high blood pressure in the pulmonary arteries, resulting in chest pain, fatigue, and shortness of breath.
TKIs, such as imatinib (Gleevec), are particularly effective in tackling the growth and mitogenic factors that become upregulated in PAH patients. These factors contribute to the...
597
Treatment for Pulmonary Arterial Hypertension: Prostacyclin Receptor Agonists01:23

Treatment for Pulmonary Arterial Hypertension: Prostacyclin Receptor Agonists

554
Prostacyclin receptor agonists are a class of therapeutic agents integral to managing pulmonary arterial hypertension (PAH). These drugs operate by mimicking the action of prostaglandin I2, or PGI2, a naturally occurring compound in the body.
These agonists bind to the IPR receptor situated on the plasma membrane of the pulmonary artery smooth muscle cells. This binding triggers a cascade of reactions known as the GS-AC-cAMP-PKA pathway. This pathway results in the relaxation of smooth muscle...
554
Treatment for Pulmonary Arterial Hypertension: Phosphodiesterase Inhibitors01:28

Treatment for Pulmonary Arterial Hypertension: Phosphodiesterase Inhibitors

663
Phosphodiesterase 5 (PDE5) inhibitors are potent enzymes that function to hydrolyze cyclic nucleotides to their corresponding 5' monophosphates. Their unique biochemical properties have been applied in treating Pulmonary Arterial Hypertension (PAH).
Among the PDE5 inhibitors, sildenafil (Revatio) stands out as a competitive and selective inhibitor. It operates by elevating cellular levels of cGMP and augmenting signaling through the cGMP-PKG pathway, promoting vasodilation. Upon oral...
663

You might also read

Related Articles

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

Sort by
Same author

Correction to: Daridorexant: First Approval.

Drugs·2022
Same author

Gefapixant: First Approval.

Drugs·2022
Same author

Daridorexant: First Approval.

Drugs·2022
Same author

Cabozantinib plus Nivolumab: A Review in Advanced Renal Cell Carcinoma.

Targeted oncology·2022
Same author

Envafolimab: First Approval.

Drugs·2022
Same author

Correction to: Avatrombopag: A Review in Thrombocytopenia.

Drugs·2021

Related Experiment Video

Updated: Mar 1, 2026

Optimized Management of Endovascular Treatment for Acute Ischemic Stroke
09:21

Optimized Management of Endovascular Treatment for Acute Ischemic Stroke

Published on: January 18, 2018

12.7K

Cerliponase Alfa: First Global Approval.

Anthony Markham1

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

Drugs
|June 8, 2017
PubMed
Summary

Cerliponase alfa (Brineura™) offers a new treatment for neuronal ceroid lipofuscinosis type 2 (CLN2), a rare pediatric neurodegenerative disease. This enzyme replacement therapy has gained global approval for managing motor function loss in CLN2 patients.

Area of Science:

  • Biochemistry
  • Genetics
  • Neurology

Background:

  • Neuronal ceroid lipofuscinosis type 2 (CLN2) is a severe pediatric neurodegenerative disease.
  • It results from a deficiency in the enzyme tripeptidyl peptidase-1 (TPP1).
  • CLN2 symptoms include motor and language decline, seizures, ataxia, blindness, and premature death.

Purpose of the Study:

  • To summarize the development of cerliponase alfa, a recombinant human TPP1 enzyme.
  • To highlight its approval for treating CLN2 disease.

Main Methods:

  • Cerliponase alfa is administered via intracerebroventricular infusion.
  • Clinical studies evaluated its efficacy in reducing functional decline in CLN2 patients.

Main Results:

More Related Videos

Isolation, Culture, and Genetic Engineering of Mammalian Primary Pigment Epithelial Cells for Non-Viral Gene Therapy
09:46

Isolation, Culture, and Genetic Engineering of Mammalian Primary Pigment Epithelial Cells for Non-Viral Gene Therapy

Published on: February 26, 2021

3.7K
A Simplified Stepwise Approach to Echo Guidance during Percutaneous Mitral Valve Repair
08:31

A Simplified Stepwise Approach to Echo Guidance during Percutaneous Mitral Valve Repair

Published on: October 16, 2021

4.5K

Related Experiment Videos

Last Updated: Mar 1, 2026

Optimized Management of Endovascular Treatment for Acute Ischemic Stroke
09:21

Optimized Management of Endovascular Treatment for Acute Ischemic Stroke

Published on: January 18, 2018

12.7K
Isolation, Culture, and Genetic Engineering of Mammalian Primary Pigment Epithelial Cells for Non-Viral Gene Therapy
09:46

Isolation, Culture, and Genetic Engineering of Mammalian Primary Pigment Epithelial Cells for Non-Viral Gene Therapy

Published on: February 26, 2021

3.7K
A Simplified Stepwise Approach to Echo Guidance during Percutaneous Mitral Valve Repair
08:31

A Simplified Stepwise Approach to Echo Guidance during Percutaneous Mitral Valve Repair

Published on: October 16, 2021

4.5K
  • Intracerebroventricular infusion of cerliponase alfa demonstrated a reduction in the progression of functional decline.
  • The therapy received global approval for treating CLN2-related motor function loss.

Conclusions:

  • Cerliponase alfa represents a significant therapeutic advancement for CLN2 disease.
  • Its development and approval mark a milestone in treating this rare pediatric neurodegenerative condition.