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Gene Therapy Trial Astonishes!

Our latest gene therapy results show phenomenal success! Earlier this year, we shared how this MGH team proved they could bring the corrected MCLN1 gene into the necessary tissues and deep structures of the brain of MLIV mice. That expression study was a terrific first step in showing that we could insert the corrected copy of the gene into the right places. In this research, however, we next asked a more complicated question: when the corrected copy of the gene is inserted, does it improve  the health of the mouse?

In this image, the gene-corrected mice are represented by the green line and the MLIV diseased mice are represented by the red line ( blue is a control). Vertical counts are one measure of healthy mouse behavior. You can see that compared to the diseased mice in this (and in every other measure), the gene corrected mice vastly outperformed the MLIV diseased mice

These exciting proof-of-concept results catapult this project ahead toward first-in-human trials. Your donations to gene therapy keep this moving forward fast!

UPenn Orphan Disease Grant, Dr. Albert Misko, MGH

Dr. Albert Misko meets a young man with MLIV
We are delighted that the latest UPenn Orphan Disease funding recipient is Dr. Albert Misko, pediatric neurologist at Massachusetts General Hospital. Dr. Misko is vigorous in his desire to document MLIV disease in patients, whether in his clinic at MGH or by traveling to see those unable to go to Boston.  His goal is to conduct neurological evaluation of as many MLIV patients as possible, in collaboration with Dr. Anne Fulton (pediatric retinal expert, Boston Children's Hospital). This unique collaboration, which involves neurological and ocular clinical examinations as well as sedated ocular imaging, brings together for the first time top scientists interested in understanding essential brain and eye aspects of the disease. 

The FDA has explained that creating this kind of documentation of the disease in the patients is essential as part of a treatment-approvals strategy. As we grow closer to clinical trials of gene therapy and drug therapy, we are thrilled to work with doctors as committed to rare children as Dr. Misko and Dr. Fulton.

The UPenn Orphan Disease Center sponsors a Dollar for Dollar match every year: we raise $25,000 and they give us a grant for $50,000 to support Dr. Misko and researchers like him!  Want to help us? Go to TeamCureML4! Every dollar matters--twice! 
ML4 mother, Paula Kutner, proudly showing a photo of her son, the inspiration for her 2017 ride!
100% Matching Dollars for ML4 Treatment Research!

Click here to give to the coveted Million Dollar Bike Ride through the University of Pennsylvania Orphan Disease Center! Each year UPenn offers us a chance to raise money for grants in treatment-based research: if we raise $25,000, they match $ for $, to give us a grant for $50,000 to the best researcher pushing forward a treatment for ML4. This year, UPenn funding is supporting Dr. Misko's clinical study of ML4 patients to document the disease in the children so we are able to begin clinical trials for the first treatment in ML4! 

Every donation is 100% matched by UPenn! What a great way to double your donations!
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