Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a rare, progressive, and fatal condition. It primarily affects males (1 out of every 3,600 to 6,000 male births). It’s caused by a lack of the dystrophin protein which is essential for muscle stability. The lack of this functioning protein causes progressive muscle weakness. Most patients lose the ability to walk by age 10 and do not live past their mid-twenties, dying as a result of heart or respiratory failure.
Researchers are still aiming to find better treatment options for this patient population. However, one of the simpler things that needs improvement is diagnosis.
The average age of diagnosis is between four to four and a half years old. However, many diagnoses are much later than this. Being diagnosed as an infant could increase the benefits of treatments, extending the quality of life for patients. An earlier diagnosis also means patients and their families have more time to plan for their future.
PRIORITY
PTC Therapeutics has just begun a new funding program called PRIORITY to support improved clinical care for individuals living with rare diseases. They aim to fund projects which are practicable, sustainable, and innovative. In its inaugural year, the grant will focus on Duchenne muscular dystrophy. More specifically, this year its goal is to fund projects which will improve newborn screening for this disease, meaning earlier diagnosis, and better care for DMD patients.
How to apply
The 2019 PRIORITY program will accept applications from clinicians in various fields such as genetics, pediatrics, and neurology. The funding will total 500,000 dollars and will be divided among accepted applications.
The deadline to apply is March 31st, 2019 and recipients will be notified by April 2019.
Who reviews applications you may wonder? PTC Therapeutics has recruited outside experts in DMD to decide which applications are approved for funding. These experts are located all over the world.
If you have developed an innovative initiative which could improve screening for DMD, make sure to get your application in before March. PTC Therapeutics says they encourage those applying to focus on three things- that their initiative is inventive, smart, and sustainable. That is, ultimately, what they know this patient community needs.
If you have innovative project ideas for a different rare disease make sure to stay tuned to hear what this program’s focus will be in 2020 as PTC has stated PRIORITY will be an annual affair.
You can read more about this new program and the call for applications here.
