Why I Believe In Ride4Gabe
Note: Matthew Alexander is a Duchenne muscular dystrophy researcher. Today we are honored to share his story of hope for Gabe and other boys with DMD.
We are in the midst of a scientific and clinical revolution when it comes to Duchenne muscular dystrophy (DMD) screening, knowledge, and therapeutic development.
Several classes of DMD therapeutic drugs are currently either in development or in clinical trials. Newer classes of steroidal drugs are in development that have fewer negative side efforts and allow DMD boys to remain walking later into life.
A variety of animal models allow us to study aspects of DMD disease symptoms that are often overlooked with the option of generating personalized DMD models based on actual patient mutations.
An exon-skipping compound, Exondys51, is approved and there are likely other exon-skipping drugs that are currently in clinical trials that show promise. Gene therapies for DMD and other neuromuscular disorders are advancing in clinical trials at a rapid pace.
Better genetic diagnostic tools to identify complex DMD patient mutations coupled with early implementation of newborn screening for DMD in certain states allow for better diagnostics and improved patient care.
While many challenges still remain, the future for a pathway towards a long-term treatment for DMD boys remains ever bright. As we enter a new decade, the potential for shifting the outcome from DMD from a fatal disorder into a manageable one becomes closer to a reality.
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