↓
 
Some posts contain affiliate links, marked with an asterisk *
Daily Journal with Mitochondrial Myopathy

Day by Day with a Movement Disorder

My Journey with a Mitochondrial Disease - "But they that wait upon the LORD shall renew their strength; they shall mount up with wings as eagles; they shall run, and not be weary; and they shall walk, and not faint." Isaiah 40:31 KJV

  • About Me – My MITO Story
  • Privacy Policy
  • Contact Us
Home→Tags Elamipretide

Diagnosis in 2006 - Parkinson's Disease and Peripheral Neuropathy, then in 2007 - Essential Myoclonus. Finally in 2011, after a muscle biopsy, I was diagnosed with Mitochondrial Myopathy as well as Peripheral Neuropathy.

Share my journey - coping with the testing, the medicines, nutrition, digestion problems, exercise, the emotions, uncertain diagnoses and no telling what else!

Tag Archives: Elamipretide

Mitochondrial Disease Treatment in Phase 3 Trial

Day by Day with a Movement Disorder Posted on October 21, 2019 by DBDecember 9, 2020  

Elamipretide

No telling how many years away from availability this is, but it seems a mitochondrial disease treatment called Elamipretide may eventually be a treatment for Mitochondrial Myopathy. As old as I am now I can’t imagine this would be much help to me. Natural aging on top of inefficient mitochondria have taken their toll on my body. I am old, and I feel old. But it’s exciting to think that the day may come in my lifetime there will be help those younger folks who struggle with this rare disease.

Read all about Elamipretide research and progress on Mitochondrial Disease News.

Phase 3 Trial is a Big Deal

Scientists test possible drug treatments on animals before getting to human trials. Human drug trials generally go through three stages. Phase 1 trials mainly want to be sure the new treatment being tested won’t harm healthy people. They track side effects compared to dosages. People volunteer and are often paid to be test subjects for Phase 1 trials. Phase 2 involves testing actual patients to see if it works or not. And of course they are on the lookout for bad side effects, even if the drug does what it in intended to do. That means any time a treatment protocol makes it through Phase 3, there is reason for optimism. Elamipretide is currently nearing the end of its Phase 3 Clinical trial.

And it seems to be actually helping the mitochondria work better. Its like “glue” that makes better connections during the chemical reaction chain that creates energy in the mitochondria. The heart muscle is illustrated in this drawing, but my problem mitochondria are in my skeletal muscles.

Clinical Trial Process is Expensive

If you read more about the whole clinical trial process, it’s not hard to see why drug research is so very expensive. Yes, we all want medicines to be cheap and easily affordable for everyone. But pharmaceutical companies will never get rich developing treatments for what are called “Rare Diseases”. It is not cost effective to do the R&D for a drug that only a few thousand people will ever need. So they have to charge more for the every day variety of meds, like for diabetes. That’s just being financially realistic. The pharmaceutical company normally applies for Orphan Drug classification for any rare diseaae research they undertake. The government provides tax incentives and a longer time as a copyrighted drug than normal medicines. And that’s meant to stimulate more research for rare disease treatments.

Ethical Drug Pricing

The problem comes when Big Pharma gets just plain greedy. They charge for the more common drugs at extravagantly elevated prices. These prices are far out of proportion to what they need to make their share holders profits. We’ve seen this happen with Insulin.

So those of us who live with one of the rare mitochondrial diseases hope for the day when a clinical trial results in a mitochondrial disease treatment protocol. We also know that such a treatment will not be cheap. And we have to face the fact that other medicines will cost more to offset the research and development costs over years of efforts to achieve that treatment.

I look forward to reading your comments!

Posted in Medicines and Supplements, MITO | Tagged Big Pharma, Clinical Trial, Elamipretide, MITO, Mitochondrial Myopathy, phase 3 | Leave a reply

Recent Posts

  • Mitochondrial Disease Treatment in Phase 3 Trial October 21, 2019
  • God’s Healing Sunlight March 31, 2018
  • Immune System Issues, Slowly Recuperating January 25, 2018
  • Flu Shot? Yes or No? December 20, 2017
  • Nutrition Information Sources December 14, 2017

Archives

Tags

balance blood glucose brain fog care giving depression diabetes diagnosis diet difficulty walking elimination difficulties Essential Myoclonus exercise exhaustion Friday Date Day gait gas Gastroenterologist GERD God insomnia MITO Mitochondrial Myopathy Movement Disorder muscle spasms nausea Neurologist nutrition pain Parkinson's peripheral neuropathy Physical Therapy prayer prescriptions Primidone PWP Quality of Life Requip Sinemet Sleep Apnea stomach stress symptoms tremors weakness Zelepar

Our Websites

  • Dirty Butter – Cherished Memories Dirty Butter – Cherished Memories
  • Dirty Butter Plush Animal Shoppe Dirty Butter Plush Animal Shoppe
  • Plush Memories Lost Toy Search Service Plush Memories Lost Toy Search Service
  • Yesterday's Memories Yesterday's Memories

Blog Friends

  • A Catholic Life – A Family Dealing with MITO
  • Anuket's Crusade
  • Baby Food Steps
  • Gilbert Guide Blog
  • Gimp Parade
  • GodsPlans
  • Leafing
  • Life According to Liz
  • Life with Shaky
  • Living in the Slow Lane
  • Mito Families!
  • Mozart Movement
  • My Father's Hand
  • My Life as a Mighty Mito Mama
  • My Own Arcadia – Spanish Language Blog with Parkinson's Information
  • Parkinson's Straight from the Horse's Mouth
  • Parkinsonism – Road to Diagnosis
  • Patients Like Me (All Kinds of Diseases)
  • PD Plus Me
  • Princess Leah Diaries
  • Shake, Rattle, and Roll
  • Taking Baby(food) Steps
  • Today with Pokie Too and PD
  • Wheelie Catholic
  • YOPD

Mitochondrial Myopathy Resources

  • Correcting Human Mitochondrial Mutations
  • mitoACTION
  • Mitochondria Research Society
  • Mitochondrial Bottleneck Cracked
  • Mitochondrial Cytopathy in Adults
  • Mitochondrial Myopathy Disease Foundation
  • Mitochondrial Vitamin Cocktail – A Guide for Patients
  • Muscular Dystrophy Association
  • NIH – Monkey DNA Swap May Block Mitochondrial Disease
  • Overview of MELAS
  • Research Match
  • Scientific American Article about Using Glutathione as a Marker
  • UAB Researchers Explore the Mystery of Mitochondria
  • When Cells Face an Energy Crisis

Nutrition Resources

  • Keto Calculator
  • Ketogenic Diet Resources
  • Maria Mind Body Health
  • Treating Constipation without Destroying Your Gut

Products I Use

  • The Energy Blueprint
  • RubyLux NIR-A Infrared Bulb
  • Satori Qigong Flow Form

Parkinson's Disease Resources

  • Parkinson's and Movement Disorders Center
  • The Brain from Top to Bottom

Peripheral Neuropathy Resources

  • About.com Guide to Peripheral Neuropathy
  • An Algorithm for the Evaluation of Peripheral Neuropathy
  • Brain Tumor Dictionary
  • Charcot-Marie-Tooth Association CMTA
  • How to Choose and Use a Walker
  • Jack Miller Center for Peripheral Neuropathy
  • Peripheral Neuropathy Fact Sheet

Radial Neuropathy Resources

  • The Wrist Drop of Saturday Night

Subcribe to Our Feed

©2022 - Day by Day with a Movement Disorder - Weaver Xtreme Theme Privacy Policy
↑
Translate »