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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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…
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…
This story comes to us courtesy of a local Rhode Island TV station's "Small But Strong" news feature, highlighting the strength and tenacity of children. 10-year-old Zoie Mercier and her…
The National Health Service (NHS) England is facing criticism following their decision not to fund the drug everolimus as a treatment for tuberous sclerosis complex associated refractory epilepsy. In comparison,…
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…
Causation vs. correlation. What does that even mean? If you ask your friend who majored in philosophy for a semester-and-a-half in college, you’ll probably get an explanation that sounds more…
Announcement is via the Child Neurology Foundation, republished here with permission: NOMINATIONS NOW ACCEPTED FOR: IS HOPE AND IS HEROES AWARDS The Infantile Spasms Action Network will once again sponsor Infantile…
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…
I was once walking through an outdoor festival near my hometown. There was a band playing off in the distance, but I didn’t know them or the song they were…
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