Happy 4th Birthday to Karah Barry- Our Friend in the Fight inspiration

Back in July we began a new feature called Friends in the Fight to spotlight other SMA children and their families in their fight with SMA and to help raise money for the gene therapy research at OSU and NCRI. Our inspiration and first featured friend Miss Karah Barry will turn 4 years old on March 27th. Karah has been through several hospitalizations and intubations this past year and keeps on showing how courageous and brave she is. She has an incredible amount of inner strength and passion for life! Happy 4th Birthday Karah! We wish you many many more birthdays and unbirthdays to come!

Birthdays are huge milestones for kids with SMA! Most children with SMA type 1 die by their second birthday! Karah along with Madison and another soon to be featured friend Nolan Shofner have asked in lieu of birthday gifts, for donations towards saving their life and the lives of their SMA friends. Please consider making an online tax deductible donation in honor or in memory of any SMA child on our secure site igive to OSU. We have the opportunity to save many lives here but even a miracle needs a little help. Thank you!

If you are an SMA family or SMA organization and would like to join us in raising funds to get this promising gene therapy to clinical trial in the next year, please contact us at miracleformadison1@mac.com to have your child or group featured.

Research Update

Dr. Brian Kaspar from Nationwide Children's Research Institute began pre pre IND discussion with the FDA last week in regards to his promising Gene Replacement Therapy for SMA. His discussion with the FDA was very encouraging and plans continue to move forward towards a Miracle for Madison and all her SMA friends. Here is a quote from Dr. Kaspar himself:

“We had our first interaction with the Food and Drug Administration regarding our gene delivery program for Spinal Muscular Atrophy on Thursday, February 24th, 2011 in what is termed a pre-pre IND (Investigational New Drug Application). For this call, we presented pertinent information to the FDA regarding the status of our studies to date, which included pre-clinical efficacy and preliminary safety data. We were impressed with the thorough, thoughtful and expert review we received from the agency in which the FDA was positive regarding our program, providing guidance for moving our translational program forward. It is important to note that these discussions were informal and non-binding, but certainly provided positive direction for us to move towards human studies. Based on our conference call and review, we are planning to perform some further dosing and safety studies that will help guide the studies that will be required for our formal application. This was an important step in our program and we are excited to continue to advance the gene delivery program forward to the clinic.” ~ Dr. Brian Kaspar Ph.D.

Read the Columbus Dispatch article:
http://www.dispatch.com/live/content/...

We couldn't have gotten to this point without all of your support over the last 13 years! We can't stop now! It is estimated that this program will need another million dollars in funding despite all of the wonderful SMA organizations like Sophia's Cure, FSMA, FightSMA and Gwendolyn Strong Foundation who have jumped on board to help this past year.

Thank you and please continue your donations and prayers for this research and for the children living everyday with this disease.