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Gene therapy gives me the courage to hope

My son Bazi is four years old and has Duchenne muscular dystrophy. It's been over a year now, and this sentence is still extraordinarily hard for me to write.

When we got the diagnosis, my emotions shifted between paralyzing fear, incredible anger, and total numbness. Never had I been so overwhelmed or helpless in my life. I just wanted to wish away this disease, or wish it upon myself, to relieve Bazi from this threat.

At the darkest of times and when my family and I felt our most hopeless, we met Pat Furlong and Parent Project Muscular Dystrophy. They were the shoulder, the therapist, the rock and… they became the light.

As of today, we do not yet have a cure that will end Duchenne, but we do have PPMD’s Gene Therapy Initiative. And I’m so grateful that this year the Rice family is matching all donations to expand this revolutionary initiative that has the potential to help my son Bazi and the entire Duchenne community.

PPMD has the most expansive and aggressive strategy in research, care, and advocacy, led by a team of brilliant and passionate people fighting on behalf of our children every day. We feel both relieved and empowered to have these talented people on our side.


This holiday season, you can take a step closer to eradicating Duchenne and help PPMD expand this cutting-edge initiative that will continue to explore the potential of therapies like CRISPR, GALGT2, and microdystrophin gene transfer. Thanks to the Rice family’s generosity, your donation will be doubled until we hit our goal of $400,000!

Gene transfer technology has gained incredible momentum, moving at a pace that is unprecedented in Duchenne. But it will take the support of this entire community to help ensure that PPMD is able to provide researchers with the funds needed to fill critical gaps in the development pipeline so that these therapies can maintain their momentum.

As a mother, it is my job to protect my children. I could not shield Bazi from this dreadful disease, but I believe with all my heart that our courage as a community will make a difference. Please join us in the fight to end Duchenne and make your commitment to PPMD today with a donation. Remember: all donations are being doubled, so this is the best time to give.

Thank you for listening to my story and for supporting PPMD’s Gene Therapy Initiative. I wish you and your family a happy and hopeful New Year.

Sincerely,

Rasha Alnaibari
PPMD Parent

P.S. All donations made to PPMD’s Gene Therapy Initiative will be matched, dollar for dollar until we hit our goal. Donate today to have your gift doubled.

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