CBS News Philly: Purple Day to Raise Epilepsy Awareness
March 26, 2015
CBS News Philly 3 featured a story about Kayla Taylor, a four-year-old girl with PCDH19 female pediatric epilepsy, an inherited disorder that affects steroid signaling in the brain. Kayla was diagnosed with PCDH19, a rare condition, two years ago when she started having seizures. But for weeks, doctors couldn't determine what was wrong with her and she was put in a medically induced coma. In the TV spot, Kayla's mother recalls the heartbreaking experience. Kayla was eventually diagnosed and fortunately is taking an approved anti-epileptic drug to control her seizures. Today, Kayla loves riding her bike.
Unlike Kayla, many girls with PCDH19 female pediatric epilepsy are not able to gain adequate control of their seizures with existing AEDs. Domain's portfolio company Marinus Pharmaceuticals, is developing ganaxolone as a potential treatment for PCDH19 female pediatric epilepsy. Ganaxolone mimics a steroid that the brain normally produces to control overexcited neurons. By targeting the same mechanism, ganaxolone may offer relief for epilepsy. Marinus is currently testing ganaxolone in a proof-of-concept Phase 2 clinical trial for PCDH19 female pediatric epilepsy and a Phase 3 clinical trial for epilepsy in adults.
Kayla's mother and their friends at PCDH19 Alliance, a patient advocacy group, hope that sharing Kayla's story will help other families looking for answers.
Learn more about Marinus' ganaxolone clinical trial for treating PCDH19 female pediatric epilepsy:
Marinus initiated the clinical trial on February 3, 2015. Read the full press release here.
FDA granted Orphan Drug Designation ganaxolone on March 25, 2015. Read the full press release here.