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…
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…
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…
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…
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…
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…
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…
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…
Marinus Pharmaceuticals has recently announced updates on its ganaxolone programs for tuberous sclerosis complex (TSC), PCDH19-related epilepsy (PCDH19-RE), and CDKL5 deficiency disorder (CDD). Ganaxolone Ganaxolone is a positive allosteric modulator…
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A recent article published in Globe Newswire heralded Marinus Pharmaceuticals’ new drug, ganaxolone. The drug has been studied in over 1600 patients and is now being tested in two clinical…
The 2019 International Tuberous Sclerosis Complex Research Conference "Changing the Course of TSC" Toronto, Ontario Are you a tuberous sclerosis patient or medical expert? Well don’t miss this important research…
The story starts in a Ana Alvarez's flat in Lima, Peru. Soon after, Ana will convert this apartment into a marijuana laboratory and underground medical center. The public will largely sympathize…
The FDA makes decisions that determine the lives and treatments of patients with rare diseases. Many of these people who make these decisions aren't impacted by the disease in their…
Elliott Cunnew, from Collaroy, Australia is what you might call a cool dad. Elliot is the father two kids, 20-year-old Amelia, and 17-year-old Lachie. During the day, he sells private…
Learning as much as you can about your diagnosis is the first step toward empowering yourself to take control of your life and make decisions that will help you live…
Abigail, a fifth grade student, originally took a trip to the Capitol to advocate for more research on tuberous sclerosis complex (TSC). The trip had unexpected results. As the Daily…
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