Sunday, July 21, 2019

Gene Therapy

The dream of a treatment for SMARD becoming available in Nate's lifetime has always felt like just that - a dream. Because the patient population is so small, research funding is very limited and there hasn't been much hope for treatment, so we've just focused on keeping Nate safe and happy in the present and have done our best to not worry too much about life down the road.
But at the moment, a possible therapy is in the pipeline at Nationwide Children's Hospital and it's kind of hard to ignore. This gene therapy has already been developed for other diseases and the same type of therapy is now being created specifically for SMARD. The therapy aims to replace the faulty gene with a healthy copy by delivering a one-time dose of a "virus" that carries the healthy gene. The results of this kind of treatment in other neuromuscular diseases have been pretty amazing. Gene therapy has potential to stop the progression of the disease and even to help kids reclaim some of the strength that they've lost. Nate's decline has been so gradual that it has been almost imperceptible even to us until we look back at pictures from years ago and notice him doing things that he'd never be able to do now. Even stopping further degeneration would be life-changing for him. 
Nationwide is already well into their research studies for SMARD and if they stay funded, clinical trials for gene therapy will start as soon as next year. But that's a big if - they are relying heavily on donations to make this happen. The nonprofit Smash SMARD has been working closely with researchers at Nationwide and have raised over $500,000 in the last few months which will be used to fund this research. A treatment is closer than it's ever been, but we've got to keep the project alive. 
Over half of the money raised so far has been from Facebook donations, and most of those donations are small amounts from people just chipping in whatever they can. So, whatever you can give really does make a difference! We still have a long way to go - almost $1,000,000 to take the project to the finish line, but I think we can do it!! It's so easy to donate on Facebook - just visit this page and click the blue "Donate" button at the top. No one sees how much you donate except the owner of the page (not me!) so we invite you to just do whatever you can, no judgement.

For those of you who want to go the extra mile, it's really easy to set up your own birthday fundraisers on Facebook for Smash SMARD. Let me know if that's something you'd want to do and I can help point you in the right direction.
You can learn more about the therapy and the work Smash SMARD is doing at
We've got a spark of hope for the first time ever, and we are so grateful to all of you who have already donated and those who have yet to donate!! 


  1. Late in the 19th century it was pocket and chatelaine watches that were most popular, but Louis replica Cartier watches saw a future in timepieces that could be worn on the wrist.Invest in an heirloom to cherish forever with a timepiece from the replica Ballon Bleu de Cartier watches watch collection.A fusion of cutting-edge technology and heritage craftsmanship, Cartier’s fine watches carry the prestige of the brand’s illustrious history and exceptional quality.

  2. International Watch Company, or IWC, was first introduced to the world in 1868 when an American Engineer,replica watches sale Florentine Ariosto Jones, decided to open his own factory in Switzerland. replica IWC watches has earned its reputation as one of the world's finest manufacturers of Swiss luxury watches. IWC only manufactures automatic and manual timepieces which include features such as the chronograph and GMT dual time zone. IWC has definitely made its mark in the world of watches with its dedication to excellence in form and function. Using a range of supplies a large selection of IWC watches at the best sale prices online so buy yours today!

  3. Various holistic approaches make alternative health therapies very effective. There are hundreds of universities and research centers that study alternative medicine and therapies.