2016

Funding of Project Goldilocks

“Lucky” is not the first word that pops to mind when we think of kids with Duchenne. But Zubin and boys like him are actually fortunate that there are multiple new therapies being developed to treat this awful disease. The fact that so many new treatments are now being developed presents us with a problem, albeit a good one to have. How do we determine which promising treatments – and combinations of treatments – should advance to clinical trials? Testing a new drug in a series of clinical trials as required by the FDA is extremely resource intensive, so it is not feasible to advance every potential therapy into the clinic. However, current animal models do not closely mirror the human form of DMD, so they aren’t great at predicting how a drug will behave in patients with Duchenne.

“Project Goldilocks” is a program designed to vet a new mouse model and testing protocol that will hopefully help our community make more informed decisions about which therapies should be advanced to human testing. Current animal models have strains of DMD that are either too severe or too mild, making it difficult to predict how a drug or combination of drugs will affect a person with Duchenne. What’s more, current testing protocols take a lot of time – a resource that families with DMD cannot spare. It is our hope that the model and series of tests being evaluated through Project Goldilocks will be “just right.” The studies are being conducted at Jackson Laboratories, one of the foremost animal testing facilities in the world. Ultimately, this work will help ensure that we utilize wisely our most precious finite resources of time, money and — most important — patients.

2015

Funding of HALO Trial

Zubin's Wish funded $100,000 in 2015 for the HALO trial, an exciting and innovative trial to explore an investigational drug that has great promise for delaying and reversing the life threatening muscle fibrosis of DMD. Along with Zubin's Wish, the fundraising community has come together to make this grassroots trial a reality. More info can be found here. By supporting Zubin's Wish, you help us make real change like this! (Note: Trial currently on hold, will update when trial resumes. In the meantime, we send our best wishes to all families in the trial that their boys can continue the medication soon.)

We have also teamed up with Charley's Fund, another organization with the same mission-to put money into translational research towards a cure for Duchenne Muscular Dystrophy. They have worked tirelessly since 2004 to fund ongoing research and promote new research towards this mission. We are honored to help them with their efforts. Below is a list of projects which you can help us fund in collaboration with them.

Funding of Pilot Trials Now

For more information on this new project, please click here.

2012

Joined Duchenne Alliance

Zubin's Wish was proud to join the Duchenne Alliance, an alliance of independent non-profit organizations dedicated to defeating Duchenne muscular dystrophy. The Duchenne Alliance member foundations collaborate to co-identify, co-review, and co-fund the most promising biomedical research.

Children with Duchenne muscular dystrophy are in dire need of our help – muscle wasting robs them of life as they generally require a wheelchair by 11 and succumb to respiratory or cardiac complications by 20. Meaningful therapies are in reach, but we need financial support to develop them quickly. Approximately one quarter of all Duchenne cases are sporadic, so it can strike any family, regardless of prior history. Since Duchenne is the most common monogenic disorder, successful therapies will have a profound impact on society.

Foundations are a vital component of the biomedical enterprise. As patient advocates, they identify the areas of greatest need in society and focus the attention of scientists on the most important problems. The DuchenneDashboard enables greater collaboration between the foundations dedicated to conquering Duchenne. As a result, each of the participating foundations can make well-informed spending decisions and are rewarded for moving good science forward.

Corporate sponsors can take great pride by directly supporting the most innovative foundations in the world. Optimized vetting by the foundations brings greater precision to therapeutic development and has had a profound impact on productivity. World-leading scientists and foundations have notably improved the speed and effectiveness of funding.

Prior to 2011

Funding of Sidenafil Trial

Our first funding commitment was a trial on the use of sidenafil for improved cardiac function in boys with DMD carried out at Kennedy Kreiger Institute which enrolled 17 boys. We were honored to help with this funding.