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The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
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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.
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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.
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[A long-awaited - but prohibitively expensive - therapy].

Bertrand Jordan1

  • 1Biologiste, généticien et immunologiste, Président d'Aprogène (Association pour la promotion de la génomique), 13007 Marseille, France.

Medecine Sciences : M/S
|February 17, 2023
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Summary

Gene therapy for hemophilia B shows long-term effectiveness after FDA and EMA approval. High costs present challenges for patients and healthcare systems.

Area of Science:

  • Biotechnology
  • Hematology
  • Genetic Medicine

Context:

  • Gene therapy development for hemophilia B has advanced significantly.
  • Recent approvals by FDA and EMA mark a milestone.

Purpose:

  • To evaluate the long-term effectiveness and durability of gene therapy for hemophilia B.
  • To discuss the implications of the high cost of this novel treatment.

Summary:

  • A novel gene therapy for hemophilia B has demonstrated sustained efficacy and durability over a three-year period.
  • The treatment, now approved by major regulatory bodies, offers a new therapeutic option.

Impact:

  • The high cost of gene therapy poses significant challenges for healthcare system accessibility and patient affordability.

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