Patient Worthy's award-winning podcast "Wait, How Do You Spell That? A Rare Disease Podcast" is back with a new episode. This week, Colby is sitting down with Kari Rosbeck, the…
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,…
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…
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…
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…
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.…
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…
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…
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…
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…
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 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,…
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…
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…
The month of May is recognized as Tuberous Sclerosis Complex Awareness Month, a time for helping to spread awareness about tuberous sclerosis complex (TSC) among the general public and in…
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…
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 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…
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…
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…
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…
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…
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…
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…
Timber Pharmaceuticals focuses on rare diseases of dermatologic origin. These include scleroderma, congenital ichthyosis, tuberous sclerosis complex, and facial angiofibromas. They are currently working on two clinical trials. Trial 1…
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