NEWPORT BEACH, Calif.--(BUSINESS WIRE)--It has taken decades, millions of research dollars, heartache and hopefulness to reach a critical milestone of the first FDA approved pharmaceutical treatment for those with Duchenne muscular dystrophy. This first treatment will only help 13% of those who battle with this genetic disease, and CureDuchenne, a national non-profit dedicated to finding a cure for Duchenne, is now redoubling its efforts to find treatments for the remaining 87% of patients with Duchenne. CureDuchenne has launched the “I am the 87%” campaign to raise awareness and fund research for the 87% percent of patients without a treatment for Duchenne.
A devastating disease that causes muscle deterioration, Duchenne muscular dystrophy affects 15,000 boys in the United States alone. Most children are diagnosed by the age of 5, lose their ability to walk in their early teens, and have an average life expectancy of mid-20s.
“This first treatment for 13% of the genetic mutations of Duchenne gives us hope that we can significantly improve the lives of all those living with Duchenne,” said Debra Miller, founder and CEO of CureDuchenne, and the mother of a son with Duchenne. “Although we’ve reached a significant milestone, this first approved drug does not treat all patients and it does not address the primary cause of death, cardiac failure. We need to take action and continue to fund science until there is a cure for all those with Duchenne.”
The first pharmaceutical treatment targets exon skipping and helps stem the muscle deterioration. As Duchenne progresses life threatening heart and respiratory conditions can occur. These issues are being targeted with advancing research funded by CureDuchenne.
The path forward to help all Duchenne patients includes vital research supported by CureDuchenne. Some of those research projects include:
- Exon skipping compounds to restore dystrophin to muscle cells,
- Combinations of approved medications that are showing potential to mitigate the effects of Duchenne,
- Cardiac therapy that addresses the leading cause of death for those with Duchenne,
- Anti-inflammatory and anti-fibrosis therapies,
- Gene therapy and gene editing to replace the faulty gene, and
- A new drug that will upregulate utrophin, a protein that compensates for the lack of dystrophin.
“The science that has helped the 13% provides a springboard for research to help the 87%,” Miller added. “Our journey to 100% is not an easy one but we’ve taken great strides forward this year. We must remain focused until there is a cure for everyone effected by Duchenne.”
Contributions can be made at www.IAmThe87percent.org.
CureDuchenne is the leading organization dedicated to finding a cure for Duchenne, the most common and most lethal form of muscular dystrophy. As the leading genetic killer of young boys, Duchenne affects more than 300,000 patients worldwide, most of them boys and young men. CureDuchenne has garnered international attention for its efforts to raise funds and awareness for Duchenne through venture philanthropy. CureDuchenne Cares provides education on best standards of care for families of Duchenne patients and healthcare professionals that treat them. www.CureDuchenne Cares.org. For more information on how to help raise awareness and funds needed for research, please visit www.cureduchenne.org, and follow us on Facebook, Twitter, Instagram and YouTube.