![]() ![]() Susan Girardin on #Take6ForPD: CIRM Board member David Higgins shares why Parkinson’s awareness matters How CIRM-funded research is bringing a lifesaving gene therapy to a 5-year-old.Apply now! Translational research funding, applications due June 13th.CIRM team shines at ASGCT annual meeting in sunny Los Angeles.Finding a treatment for Tay-Sachs disease. ![]() Apply now! Translational research funding, applications due June 13th.Smoking marijuana could be bad for your heart, but there is an unusual remedy.More Photos Categories Categories Archives Archives Top Read Posts Learn more about CIRM’s commitment to fighting rare conditions like SCID and about our commitment to advancing new therapies for prevalent diseases like cancer, HIV/AIDS and stroke. These efforts will ensure that more patients like Seersha will have access to lifesaving treatments through clinical trials. That includes launching a Patient Support Program, which will provide resources and financial support to California patients being evaluated or enrolled in CIRM-funded clinical trials, with a particular emphasis on helping underserved populations. Part of our investment in rare diseases includes developing infrastructure programs to promote their access and affordability to diverse communities. In addition to funding research and clinical trials for prevalent diseases, CIRM recognizes the importance of finding treatments for rare condition such as SCID, retinitis pigmentosa, sickle cell disease, Huntington’s disease, and Duchenne muscular dystrophy. “This agreement with the Bespoke Gene Therapy Consortium will enable us to be part of a bigger partnership, one that can advance the field, overcome obstacles and lead to breakthroughs for many rare diseases,” Millan added. CIRM funding can then support the IND-enabling research, manufacturing and clinical trial activities of these programs. That’s why last year, CIRM signed a Memorandum of Understanding (MOU) with the Foundation for the National Institutes of Health (FNIH) to join the Bespoke Gene Therapy Consortium (BGTC), a public-private partnership managed by FNIH that brings together multiple public and private sector organizations to streamline the development and delivery of gene therapies for rare diseases.Īs part of the Consortium, CIRM will identify specific rare disease gene therapy research programs in California that are eligible to be part of the AMP BGTC. “But even an agency with our resources can only do so much.” “At CIRM we have funded several projects using gene therapy to help treat, and even cure, people with rare diseases such as SCID,” says Dr. That equates to 1 in 10 Americans suffering from conditions for which limited treatment options exist. “I don’t like the word ‘normal’, but I’m ready to have something normal for her,” her mom told CNN.Īccording to the National Institutes of Health (NIH), approximately 7,000 rare diseases affect between 25 and 30 million Americans like Seersha. Seersha’s journey was recently featured in CNN and is a true testament to the power of partnering to advancing medical research, including for rare diseases. Now, thanks to the work of researchers at UCLA Broad Stem Cell Research Center, a group of dedicated parents at SCID Angels for Life Foundation and funding from California Institute for Regenerative Medicine (CIRM), Seersha is close to receiving a lifesaving gene therapy treatment that will correct the defective gene disabling her immune system. The five-year-old was born with ADA-SCID, a condition so rare that only eight babies a year are born with it in the United States. For her first year of life, Seersha Sulack stayed mostly in her bedroom because something as simple as a common cold could have killed her. ![]()
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