Charlotte, NC Declares May as Tuberous Sclerosis Complex Month
When Kristen Moore’s son Jackson was first diagnosed with tuberous sclerosis complex (TSC), she decided to translate her emotions into action. As WSOC-TV reports, she dove into education, awareness,…
Girl with TSC Shows no Symptoms After Clinical Trial Involvement
When Cheryl Navar decided to have a child, in-vitro fertilization (IVF) seemed like the right move. Navar knew that she had a cancer gene and didn’t want to transfer…
“We May Bend, But Never Break”: How the Chandler Family Faces a Tuberous Sclerosis Complex (TSC) Diagnosis with Resilience
Gavin Chandler loves toy cars, watching Big City Greens, fans, the “Baby Shark” song, and anything that lights up or makes noise—including, his mother Jessica shares, the vacuum cleaner. He…
Study of the Week: Research Using “Mini-Kidneys” Leads to Tuberous Sclerosis Complex Discovery
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Epilepsy Research UK Joins Tuberous Sclerosis Association in Funding Collaboration
According to a recent announcement, Epilepsy Research UK has announced a funding collaboration with the Tuberous Sclerosis Association which will support the investigation of tuberous sclerosis complex (TSC)-associated epilepsy.…
Researchers Created Mini-Kidneys to Identify Tuberous Sclerosis Complex and Solve a Medical Mystery
Tuberous sclerosis complex (TSC) is classified as a rare genetic disorder that causes typically benign tumors in the brain, skin, heart, kidneys, or lungs. As described in a recent article…
Family Participated in “Step Forward to Cure TSC” to Raise Awareness
When Hailey Huddleston was just two years old, she had her first seizure. At first, her parents – Tim and Lisa – weren’t sure if Hailey was having a night…
Facial Angiofibroma Associated with Tuberous Sclerosis Complex Now Has an FDA Approved Topical Therapy
Nobelpharma has just announced the FDA approval of sirolimus .2% gel (called Hyftor). It is now the first approved, topical therapy for facial angiofibroma associated with the rare condition tuberous…
Rare Classroom: Tuberous Sclerosis Complex
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
The TSC Navigator: A New Resource to Guide Families through the TSC Journey (Pt. 2)
In Part 1 of "The TSC Navigator: A New Resource to Guide Families through the TSC Journey," I sat down with Ashley Pounders to discuss the TSC Navigator, its purpose,…
The TSC Navigator: A New Resource to Guide Families through the TSC Journey (Pt. 1)
The TSC Alliance® first began as a grassroots organization, founded by motivated mothers whose children had been diagnosed with tuberous sclerosis complex (TSC), a rare genetic disease. Since its founding,…
Phase 2 Trial Data Available on Ganaxolone for TSC
According to an August 17, 2021 news release from pharmaceutical company Marinus Pharmaceuticals, Inc. (“Marinus”), data is available from a Phase 2 clinical trial evaluating oral ganaxolone for patients…
Epidiolex Approved to Treat TSC-Related Seizures
It’s no secret that cannabis and its components are highly controversial. However, cannabis has been used to treat a variety of conditions, including seizures. According to Candid Chronicle, another related…
Raising Children with Tuberous Sclerosis Complex (TSC)
Not many people expect a rare disease diagnosis out of the blue. For many, they had been seeking the correct diagnosis for years before finally being given the name of…
Brother with TSC Motivates Harvard-Bound Sister
Chiprez Ramirez describes her younger brother Francisco as her "little ray of sunshine." Talking to MSN, she said that his rare disease journey and strength are what motivate her to…
Jaxson’s Story: Living with Tuberous Sclerosis Complex (TSC)
Jaxson Corcoran was born with tuberous sclerosis complex (TSC), a rare genetic disorder that is characterized by benign tumors forming throughout the body. Treating this condition has become a family…
Epidyolex Approved in Europe for TSC
Did you know that there are over 80 chemicals, called cannabinoids, that can be sourced from the Cannabis sativa plant (marijuana or hemp)? Now, Europe is harnessing this for…
Metformin Benefits Patients with Tuberous Sclerosis Complex
Could repurposing certain drugs help treat other conditions? Well, according to Technology Networks, yes. Recently, researchers from the UCL Great Ormond Institute of Child Health and the Great Ormond Street…
COVID-19 Didn’t Slow Down the Tuberous Sclerosis Alliance
The ongoing COVID-19 pandemic has thrown the world into chaos. With over 500,000 dead in the US alone, the impacts of this global health crisis have affected many different facets…
Study Reveals Gene Therapy as a Possible Tuberous Sclerosis Complex Treatment
According to a story from news-medical.net, a team of scientists affiliated with Massachusetts General Hospital has conducted a study revealing that gene therapy could be an effective treatment for tuberous…
New Study Aims to Improve Outcomes for Tuberous Sclerosis Complex Patients
Tuberous sclerosis complex (TSC) is a rare disease that, like many other rare diseases, is not yet fully understood by medical professionals. Because of this, it is very difficult to…
FDA Approves the Cannabis Based Drug Epidiolex to Treat TSC Seizures
As published by the Food and Drug Administration; The U.S. FDA has just approved the first FDA approved therapy derived from cannabis (Epidiolex, a CBD solution) to treat tuberous sclerosis…
A Possible Treatment for CDKL5 Deficiency Disorder has Earned Rare Pediatric Disease Designation
According to a story from BioSpace, the pharmaceutical company Marinus Pharmaceuticals, Inc. announced recently that its investigational product candidate ganaxolone has earned Rare Pediatric Disease designation from the US Food…
Tuberous Sclerosis Complex: CBD Oral Solution Epidiolex Approved to Treat Seizures
According to a story from GlobeNewswire, the biopharmaceutical company GW Pharmaceuticals plc recently announced that its CBD oral solution Epidiolex® has recently been approved as a treatment for seizures associated…
