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…
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…
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…
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…
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 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…
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…
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…
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…
Not only are rare diseases well, rare, but the same rare disease can also affect different patients in a multitude of ways. For instance, some diseases can impact many different…
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…
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 U.S. Food and Drug Administration (FDA) have just approved a drug called Afinitor DISPERZ for treating tuberous sclerosis complex-associated partial onset seizures, reports PR Newswire. This makes it the…
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