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.
Wednesday, March 2, 2011
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