Please enjoy the video....it took ALL DAY! After the video I have some exciting news to share....
I am excited to share with my family and friends the information about the promising research being done in Dr Kaspars lab. Every night we say prayers with Larkin and in my prayer I always say, "please help the researchers find a cure for SMA...give them the willpower, strength and wisdom to find a cure". I think with the MANY prayers from all our SMA family/community God is answering them. I'm so hopeful, excited and most of all THANKFUL! Here is the article from the Sophia's Cure Foundation's Facebook page.
*BREAKING NEWS*
The final FDA guided studies have begun. The first cohort of mice have been injected with scAAV9. The injections were a success. We are extremely excited to be in the final stages moving this program towards a human clinica...l trial application for Spinal Muscular Atrophy.
The Kaspar Laboratory at The Research Institute at Nationwide Children's Hospital and The Ohio State University in Columbus, Ohio recently initiated a formal FDA (Food and Drug Administration)sanctioned toxicology and biodistribution studies at BioReliance, a Contract Research Laboratory in the Washington D.C. area for a systemic gene delivery of AAV9-SMN for the treatment of Spinal Muscular Atrophy. The purpose of these studies are to establish the safety and distribution profiles required to advance to human clinical studies, known as a Investigational New Drug application. These studies were outlined to the FDA in a pre-Investigational New Drug Application and designed to meet all of the requirements to enter human clinical studies for treating SMA patients. The studies involve a 6 month evaluation of the gene delivery in mice. Dr. Kaspar stated, "This marks an important milestone in our program to advance a systemic gene therapy for SMA to the clinic, Our team has advanced this program at a remarkable pace and we are very pleased with our studies that show we can target motor neurons efficiently as well as a very clean safety profile to date." Drs. Foust and Kaspar initiated these pivotal studies at BioReliance by injecting the animals for these safety studies through funding by Sophia's Cure Foundation. Kaspar notes, "We are truly thankful for the support of the SMA community and the committment from Sophia's Cure Foundation to see this gene delivery program to this stage. My team and I are committed to advancing this highly promising therapeutic to the clinic." Over the course of the 6-month study, there are many processes to advance, including making the clinical grade gene therapy and compliling the complex data package to submit to the FDA. "We're excited and working non-stop with an amazing group of highly committed and talented researchers and clinicians to see this to clinical reality. We're thankful for everyone's committment to helping us. including our researchers and the SMA community at large", Kaspar noted.
The final FDA guided studies have begun. The first cohort of mice have been injected with scAAV9. The injections were a success. We are extremely excited to be in the final stages moving this program towards a human clinica...l trial application for Spinal Muscular Atrophy.
The Kaspar Laboratory at The Research Institute at Nationwide Children's Hospital and The Ohio State University in Columbus, Ohio recently initiated a formal FDA (Food and Drug Administration)sanctioned toxicology and biodistribution studies at BioReliance, a Contract Research Laboratory in the Washington D.C. area for a systemic gene delivery of AAV9-SMN for the treatment of Spinal Muscular Atrophy. The purpose of these studies are to establish the safety and distribution profiles required to advance to human clinical studies, known as a Investigational New Drug application. These studies were outlined to the FDA in a pre-Investigational New Drug Application and designed to meet all of the requirements to enter human clinical studies for treating SMA patients. The studies involve a 6 month evaluation of the gene delivery in mice. Dr. Kaspar stated, "This marks an important milestone in our program to advance a systemic gene therapy for SMA to the clinic, Our team has advanced this program at a remarkable pace and we are very pleased with our studies that show we can target motor neurons efficiently as well as a very clean safety profile to date." Drs. Foust and Kaspar initiated these pivotal studies at BioReliance by injecting the animals for these safety studies through funding by Sophia's Cure Foundation. Kaspar notes, "We are truly thankful for the support of the SMA community and the committment from Sophia's Cure Foundation to see this gene delivery program to this stage. My team and I are committed to advancing this highly promising therapeutic to the clinic." Over the course of the 6-month study, there are many processes to advance, including making the clinical grade gene therapy and compliling the complex data package to submit to the FDA. "We're excited and working non-stop with an amazing group of highly committed and talented researchers and clinicians to see this to clinical reality. We're thankful for everyone's committment to helping us. including our researchers and the SMA community at large", Kaspar noted.
