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Gene Therapy Guides Future Treatment for Rare Diseases

Of the numerous, notable medical breakthroughs in the last decade, few have elicited more genuine hope than the promise of gene therapy. The cost, time, and effectiveness of current treatments for rare conditions have evolved significantly, but the vast majority still have no cure, making this a challenging journey to navigate. So it is truly gratifying to realize the enormous progress that’s been made and continues to be made, in replacing the need for surgery and/or a lifetime of a regimen of drugs with highly advanced, singular techniques centered on inserting a gene into a patient’s cells.

Access the Gene Therapy Whitepaper

Researchers are testing a variety of approaches that include: replacing a mutated gene that causes disease with a healthy copy of the gene, inactivating a mutated gene that is functioning improperly or introducing a new or modified gene into the body to help fight a disease. Gene editing technology enables targeted changes in the genome, such as fixing a mutation or removing or adding a gene. Gene-targeted therapy refers to a treatment that alters genetic activity using a variety of therapeutic agents, such as exon skipping drugs or gene silencing molecules. While these therapies target somatic cells, making them corrective to the patient only and not the next generation, they are poised to dramatically alter the entire landscape of best practices in treating rare diseases by offering a cure rather than symptom management.

As Founder and CEO of a specialty pharmacy dedicated to providing comprehensive therapies for a number of uncommon, often inherited conditions, I am inspired by the burgeoning pipeline of gene therapy trials and their life-changing potential for patients. In this whitepaper, I highlight a few and share my perspective on where we’ve been and most importantly, where we’re going.

Access our latest whitepaper and get a detailed personal perspective on how gene therapies are guiding the future of treatment for the following rare diseases:

  • Duchenne Muscular Dystrophy (DMD)
  • Hereditary Angioedema (HAE)
  • Idiopathic Pulmonary Fibrosis (IPF)
  • Spinal Muscular Atrophy (SMA)
  • Gaucher’s Disease Type 1
  • Mucopolysaccharidosis (MPS)
  • Fabry Disease
  • Pompe Disease

Click here to access your digital copy of the whitepaper, “Gene Therapy Guides Future of Treatment for Rare Diseases.”

Author: Mike Fieri, is the Founder & CEO of Orsini Specialty Pharmacy, the leading independent specialty pharmacy focused on rare diseases and gene therapies. Mike has 30 years of specialty pharmacy experience focused on strategic and business development endeavors within corporate and new enterprises.