On March 9-10, 2023, Dynamic Global Events hosted the virtual Global Innovation in Patient Advocacy conference. This event was geared towards various stakeholders in the rare disease space and was…
Before you read on, don't forget to check out Part 1 of the story. In Part 1, Whitney talks about what CDKL5 deficiency disorder (CDD) is and the diagnostic journey for her…
A year after her daughter Havilah was diagnosed with CDKL5 deficiency disorder (CDD), Whitney Mitchell’s pediatrician looked her in the eyes and said something that Whitney still carries with her…
At just two days old, Leita’s son Glyn started having infantile spasms. For the next sixteen years, Glyn’s seizures worsened. Leita also had two other children – twins named Asha…
According to a recent article in Businesswire, The Lancet has published data from the third phase of the Marigold trial. This clinical trial evaluated ganaxalone - marketed as ZTALMY - as a…
Until recently, patients with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) did not have an FDA-approved treatment option. This all changed when the regulatory agency granted approval to ZTALMY, also…
In a late October news release from pharmaceutical company Marinus Pharmaceuticals, Inc. ("Marinus"), the company shared that it would be collaborating with the Loulou Foundation, a private UK nonprofit dedicated…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Currently, there are no known cures for CDKL5 deficiency disorder, a rare genetic disorder characterized by seizures and severe developmental delays. Treatment, rather, is symptomatic and supportive. However, a news…
The month of June is recognized as CDKL5 Awareness Month, a time set aside to spread awareness about CDKL5 deficiency disorder among the general public and the medical community. June…
On January 13, 2021, pharmaceutical company Marinus Pharmaceuticals, Inc. ("Marinus") shared via press release that the FDA gave the company a positive response following efficacy and safety data from the…
Developmental and epileptic encephalopathies (DEEs), or rare epilepsies, are somewhat of a newer topic. However, researchers are already making strides in creating treatments to address unmet patient needs. On…
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…
June 17th is recognized as World CDKL5 Awareness Day and the month of June is considered CDKL5 Awareness Month. First organized by the International Foundation for CDKL5 Research (IFCR), the…
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…
A new study has been initiated by a digital health company in the United Kingdom called Congenica Ltd. This study focuses on re-annotating genes that may be associated with…
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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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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A recent announcement by Atomico, one of Europe’s largest venture capital firms based in London, introduced its new partner Healx. The startup can boast of having the world’s most comprehensive…
According to a story from sectorpublishingintelligence.co.uk, the drug company Ovid Therapeutics Inc. has recently announced that the early data from its phase 2 clinical trial is mostly encouraging. This clinical…
Extended Collaboration Amicus Therapeutics has just announced that they are expanding their collaboration with the University of Pennsylvania's Perelman School of Medicine for the next five years. This collaboration is…
Confusion, nausea, fever, anger, then back to normal: this was Lauren Vandiver’s life beginning in early childhood. Her parents, Phillip and Angela Vandiver, spent more than twenty years asking the…
According to a story from the Greenock Telegraph, twelve year old Megan McGarva, who was born with CDLK5 deficiency, will soon have to face a serious spinal operation. She has…
According to a story from globenewswire.com, the biopharmaceutical company Marinus Pharmaceuticals, Inc., recently announced the presentation of Phase 2 clinical trial of the drug ganaxolone as a treatment for CDKL5…
According to a story from pm360online.com, the biopharmaceutical company Ovid Therapeutics recently announced that it had received the 2018 Company Making a Difference Award from the CDKL5 Forum.This recognition was given to…
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