Brighter days to come.
duchenne muscular dystrophy
Duchenne Muscular Dystrophy (DMD) is a lethal genetic childhood disease.
DMD causes muscles to become weak and damaged over time, and is eventually fatal. The genetic mutation that causes DMD happens before birth and can be inherited, or new mutations in the gene can occur spontaneously. It primarily affects males, but, in rare cases, can also affect females.
Currently, about 15 children in our community suffer from this devastating disease. We invite you to learn more about DMD, and how you can help.
Suneel's Light Funds Research Leading to First FDA-Approved Drug for DMD
The US Food and Drug Administration (FDA) approved the first drug, produced by Sarepta Therapeutics, to treat Duchenne Muscular Dystrophy (DMD) patients in September 2016. Exondys 51 (eteplirsen) gained accelerated approval to treat DMD patients with a confirmed mutation of the dystrophin gene amenable to exon 51 skipping to improve muscle and motor function. While this particular mutation affects only about 13 percent of the DMD patient population, Exondys 51 is now the first therapy approved by the FDA that is specifically indicated to treat this disease.
Making Life-Changing Research a Reality
Suneel’s Light provided research funding to Akashi Therapeutics for the development of a drug that would alleviate the effects of the muscle damage in DMD. HT-100 (delayed-release halofuginone) is an orally available, small molecule developed to reduce fibrosis and inflammation and promote healthy muscle fiber regeneration in boys with DMD.
One of more than 30 forms of muscular dystrophy
Average age of diagnosis
Percent of kids in wheelchairs by age 15
Number of people it takes to make a difference (you)