WE ARE ON A MISSION TO FIND A CURE
SMARD stands for Spinal Muscular Atrophy with Respiratory Distress (also known as SMARD1). There is little known about this ultra rare and often fatal disease. Its rarity is of no comfort for the families affected by SMARD, and so we are building a community to promote awareness and research to make a cure a reality.
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SmashSMARD is proud to announce its support of a promising research program to be conducted by Dr. Kathrin Meyer at the Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio USA. Dr. Meyer's laboratory at AWRI has a longstanding translational focus on neurodegenerative diseases and an encouraging track record of moving therapeutic programs towards clinical trial.
With SmashSMARD’s funding support, Dr. Meyer is now bringing that focus to SMARD. Dr. Meyer’s lab develops Gene Therapies for neurological and neuromuscular disorders by using an approach that increases full length protein expression.
SmashSMARD looks forward to Dr. Meyer’s work in supporting the translation of SMARD research to a CLINICAL TRIAL IN THE COMING MONTHS.
We need YOUR to keep the research moving forward towards a clinical trial. To date, in partnership with smashSMARD Germany, we have already raised around $2 million, but our goal for 2020 is to raise over $250,000!!!
Gene therapy aims to replace or correct the faulty IGHMBP2 gene and is a well-documented treatment for this disease in animal models. This video shows success of gene therapy in mice at the Univesity of Milan (AAV-null mouse is untreated; AAV-IGHMBP2 has received gene therapy).
We are Funding Research Now for SMARD, CMT2S and IGHMBP2!
Our small SMARD community is committed to spreading the word and doing all we can to get these wonderful kids treatment as soon as possible. For a critically ill individual, everyday is a celebration of life and we plan to celebrate each of these faces for many, many more days and years to come.