Study Reveals Gene Therapy as a Possible Tuberous Sclerosis Complex Treatment
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Study Reveals Gene Therapy as a Possible Tuberous Sclerosis Complex Treatment

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…

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A Possible Treatment for CDKL5 Deficiency Disorder has Earned Rare Pediatric Disease Designation
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A Possible Treatment for CDKL5 Deficiency Disorder has Earned Rare Pediatric Disease Designation

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…

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Tuberous Sclerosis Complex: CBD Oral Solution Epidiolex Approved to Treat Seizures
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Tuberous Sclerosis Complex: CBD Oral Solution Epidiolex Approved to Treat Seizures

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…

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Congenital Ichthyosis and Tuberous Sclerosis Complex Clinical Trials are Now Enrolling Patients
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Congenital Ichthyosis and Tuberous Sclerosis Complex Clinical Trials are Now Enrolling Patients

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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Ganaxolone Being Investigated as Treatment for Tuberous Sclerosis and CDKL5 Deficiency Disorder
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Ganaxolone Being Investigated as Treatment for Tuberous Sclerosis and CDKL5 Deficiency Disorder

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…

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Rare Epilepsy Updates:  One New Drug, Two Clinical Trials, and Orphan Drug Designation

  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…

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