TSC Matters

Archive for October, 2019

TSC Matters is a bimonthly e-newsletter featuring news from the TS Alliance, TSC research updates, upcoming TS Alliance events and more! Subscribe to stay up-to-date on everything happening at the TS Alliance.

Engage Therapeutics Clinical Trial

Engage Therapeutics is a small biotech company working to improve life with epilepsy. Toward this end, they are developing an inhaler rescue therapy that stops seizures in their tracks. If you have frequent, predictably-patterned seizures, you may qualify for a paid trial of this product. Visit epilepsyhealthstudy.com.Read More

Update on State Funding in Maryland and Missouri

The Missouri State Legislature appropriated $250,000 in the state’s 2020 budget for the TSC Center of Missouri. In addition, $500,000 for the new TSC Center of Maryland was appropriated in Maryland Governor Larry Hogan’s 2020 budget and approved by the Maryland General Assembly with the support of Senator Brian Feldman and Delegate Nicholaus Kipke. Thank you to all those who made these appropirations possible, both were the result of incredible advocacy efforts in Missouri and Maryland. Read more about these appropriations in the latest Perspective Magazine Read More

Upcoming Community Events

TSC Family Fun Day at Pullen Park Sunday, October 20, 2019 Pullen Park; Carousel Shelter 520 Ashe Ave Raleigh, NC 27606 Questions and RSVP: Karen Johnston at karen.m.whittington@gmail.com or (919) 451-1842   Southern California (Teen & Adult)  Evening of Bowling Sunday, November 17, 2019 Pins & Pockets Entertainment 32250 Mission Trail Lake Elsinore, CA 92530 Please RSVP by November 12 to Tara Palage at tara.palage@yahoo.com or (619) 977-4114Read More

TSC Clinic Experience of Care Survey

The TSC Alliance would like to know about your experience at the TSC Clinic you/your child go to for care.  Your comments in this online survey will be shared with the TSC Clinic Director and staff.  The 5-minute survey is anonymous – your answers will not be linked to any information that can identify you.   We invite you to complete this survey any time after you’ve been to the TSC Clinic. If you have any questions, contact Jo Anne Nakagawa at jnakagawa@tscalliance.org. Take the SurveyRead More

Phenotypic Variability in Tuberous Sclerosis Complex (TSC)

Dr. Narayanan and colleagues at the Translational Genomics Research Institute (TGen) in Phoenix, Arizona are conducting a research study aimed at identifying genetic modifiers of disease severity in TSC.  For this study, we are recruiting families with children of all ages in which a parent and child both have TSC.  In particular, we are looking for those families where the parent has little or no neurological problems (mild disease) and the child has severe neurological disease (severity assessed by scoring epilepsy and cognitive symptoms).  Our goal is to identify those genetic differences between affected parent and affected child that might account for difference...Read More

Clinical Trial for Individuals Diagnosed with Tuberous Sclerosis Complex (TSC) and Cutaneous Angiofibromas

We are currently conducting a clinical trial for individuals diagnosed with TSC with facial bumps called cutaneous angiofibromas. The goal of this study is to investigate if the study drug, sirolimus, improves cutaneous angiofibromas in individuals diagnosed with TSC. The trial is sponsored by a pharmaceutical company named Aucta Pharmaceuticals, Inc, and it will involve 6 to 8 centers including Boston Children’s Hospital, Children’s Hospital of Philadelphia, Le Bonheur Children’s Hospital, Children’s Hospital Colorado, Children’s Hospital Los Angeles, and Translational Genomic Research Institute.Read More

Preventing Epilepsy Using Vigabatrin in Infants with Tuberous Sclerosis Complex (PREVeNT Trial)

Enrollment still open! Where: TSC Clinical Research Consortium sites at Boston Children’s Hospital, Cincinnati Children’s Hospital Medical Center, University of Alabama at Birmingham, University of California at Los Angeles, University of Texas at Houston, Minnesota Epilepsy Group, PA, Stanford University, Beaumont Children’s Hospital, Washington University in St. Louis, Children’s Hospital of Orange County, Children’s National Medical Center, The Children’s Hospital of Philadelphia, Duke University Health Center, and Mount Sinai Principal Investigators: Boston (Mustafa Sahin, MD, PhD), Cincinnati (Darcy Krueger, MD, PhD), Birmingham (Martina Bebin, MD, MPA), Los Angeles (Joyce Wu, MD), Houston (Mary Kay Koenig, MD), Minnesota (Michael Frost, MD),...Read More