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…
A code for an autism-linked syndrome has now been established and entered into the U.S. diagnostic manual that catalogs health conditions. According to a recent report in Market Watch,…
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…
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…
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…
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 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…
The TACERN Study Group has recently conducted a study in order to discover an algorithm that detects tubers during MRIs. Deep learning was employed in an effort to apply this…
People affected by rare diseases often find it difficult to find adequate treatment for their conditions. Due to the rarity of their diseases, there are often no doctors who specialize…
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…
Marinus Pharmaceuticals is a pharmaceutical company that works to create treatments for epilepsy and other neuropsychiatric disorders. They are in the process of conducting clinical trials to evaluate the safety…
As originally reported in MSU Today; in new experiments on mice, researchers looked into how the TS gene is related to autism. Tuberous sclerosis is a rare disorder known for…
According to a press release from the British biotechnology company GW Pharmaceuticals, the company has reported successful results at the conclusion of its phase 3 trial of tuberous sclerosis complex…
Multiple Sclerosis (MS) was long believed to be an illness that could only affect adults. In fact, the pediatric form of this disease was not discovered until just over 10…
Tuberous Sclerosis Tuberous sclerosis complex (TSC) is a rare condition which results in the buildup of tumors in the skin, kidneys, brain, as well as other organs. Although the tumors are…
2018 World TSC Conference This event will be an opportunity for tuberous sclerosis patients and their families to network with one another. Participants will also be able to learn about…
Research into possible facial creams containing the medicine rapamycin have found that they are effective for reducing facial tumours in people with tuberous sclerosis complex. The full article can be…
According to a story from edinburghnews.com, mother Karen Gray is about to seek access to cannabidiol oil for her five-year-old son, Murray, who has a rare type of epilepsy, Myoclonic Astatic…
Scientists are investigating whether delaying the onset of epilepsy in very young children could reduce or prevent other neurological conditions such as learning difficulties and autism, reports Medical Xpress. This…
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…
Kelley Fox is 23 years old, but developmentally she’s only around 5 or 6 years old. She lost half of her brain due to a disorder called tuberous sclerosis. The…
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