TSC Alliance

Government relations efforts focus on increasing federal and state appropriations for TSC research, raising awareness, and collaborating with government partners to drive TSC research forward and improve clinical care and treatment options for individuals with TSC. On the state level, the TSC Alliance advocated for state funding for TSC centers in Missouri and Maryland, resulting in at least $1.1 million in state appropriations. Additionally, Alabama held their first advocacy proclamation day with their governor the annual TSC Alliance march on capitol hill to advocate for federal funding for the Tuberous Sclerosis complex research program (tscrp) at the department of defense's (dod) congressionally directed medical research program (cdmrp) took place in early march 2022. More than 204 people from 42 states registered to participate in the virtual march on capitol hill. Advocates made virtual appointments to advocate for $10 million in funding for the tscrp. The house tscrp dear colleague letter sponsored by representatives mullin (r-ok) and raskin (d-md) closed with 205 signers. The senate letter sponsored by senators cramer (r-nd) and heinrich (d-nm) closed with 35 signers. Total funding for the tscrp has been $113 million since 2002, including an $8 million appropriation approved in december 2022 for fy23. Research performed through this program has recently led to additional clinical trials including determining if imatinib, a drug fda-approved for cancer, can safely improve levels of vegf-d, a biomarker of lymphangioleiomyomatosis (lam), a life-threatening lung manifestation of TSC, funded in fy2013; two tscrp awards in fy2012 and fy2015 that enabled generation of a potential approach for gene therapy of TSC, which has shown promising results in a mouse model of TSC tumors in the brain; a multi-site clinical trial testing the efficacy of an experimental topical rapamycin cream to treat the disfiguring facial tumors, called facial angiofibromas, caused by TSC funded in fy2010 that led the the third fda-approved drug to treat TSC in april 2022; and a clinical research network that was created to test potential new therapies, to validate biomarkers, and to learn the natural history of leading to a clinical trial funded in fy2012. Data obtained from an fy2010 tscrp clinical research award to define early autism predictors in TSC and an fy2014 tscrp award for a pilot clinical trial is being tested in a large, nih-funded clinical trial looking at the effectiveness of a behavioral intervention strategy, jasper, to improve outcomes in children with autism. The tscrp has also funded research to develop animal models of TSC that have seizures, enabling a better understanding of the etiology of TSC. Based on data from tscrp-funded animal models of TSC that have seizures and share pathology related to that of traumatic brain injury, an industry-sponsored clinical trial demonstrated the effectiveness the mtor inhibitor, everolimus, at treating epilepsy in many individuals with TSC. None of this progress would have been possible without the critical support provided through the tscrp.global outreach works to address unmet needs within the global TSC community. The program provides the opportunity for the TSC Alliance to share experiences and assist in the start-up of support of tsc-related organizations in other countries. A global Alliance is a structured group of empowered and caring volunteers who work closely with the TSC Alliance to facilitate local connections for individuals and families affected by TSC and raise revenue and awareness while supporting the mission of the organization. The TSC Alliance has six global partnerships, including TSC Alliance of israel, ts canada st, TSC Alliance of mexico, TSC Alliance foundation (in thailand), hungarian foundation for Tuberous Sclerosis, and TSC Alliance of india. The TSC Alliance recognizes 13 TSC clinics in global Alliance countries. Professional education expands programs to target researchers and healthcare providers caring for individuals with TSC, medical students, genetic counselors and educators to minimize the consequences of ignorance and misinformation. In december, therapeutic advances in rare disease published "racial differences in the dermatological manifestations of Tuberous Sclerosis complex and the potential effects on diagnosis and care," to raise awareness of dermatological manifestations associated with TSC, compare their appearance by race, and consider how recognition of these features could impact diagnosis and treatment of tsc.the TSC Alliance participated in or presented at 28 professional meetings in 2022 including tandem consortium meeting, nobelpharma town hall, mallinckrodt ad board, napnap, acmg, tand mini-symposium (tandem), muscular dystrophy coordinating committee spring 2022 meeting, american thoracic society 2022 international conference, rare diseases international: 75th world health assembly side event: networking, interagency collaborative to advance research in epilepsy (icare) annual meeting, ninds nonprofit forum, world TSC conference, lamposium, global genes rare summit, hra fall 2022 members meeting, rdcrn fall meeting, mn pharm conf, achg, global genes atlanta, isan membership meeting, everylife nbs bootcamp, tscrp, american epilepsy society, ats par planning meeting, arpa-h rare disease planning meeting, tandem south africa, ats par lung week, and pame.

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