On March 7, 2024, the United Kingdom’s Medicines and Healthcare products Regulatory Agency shared via press release that the agency has approved Ztalmy (ganaxolone) for people living with CDKL5 deficiency…
As it stands today, there are no cures for CDKL5 deficiency disorder. Treatment is symptomatic and supportive, and includes physical, occupational, and speech therapy. CDKL5 deficiency disorder causes seizures,…
June 17th is International CDKL5 Day. During the month of June let’s tag every day with a reminder to those in the CDKL5 community to promote collaboration and fundraising.…
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…
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…
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…
As originally reported in BioSpectrum; the World Health Organization (WHO) has just approved the disease code for CDKL5 Deficiency Disorder (CDD), adding it to the list used worldwide for 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…
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…
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…
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…
Takeda Pharmaceutical Company Limited and Ovid Therapeutics have released an overview of the clinical development program for TAK-935/OV935, an investigational drug that is being developed as a potential anti-epileptic…
The Orphan Disease Center in association with the University of Pennsylvania and the Loulou Foundation just announced their 2018 Pilot Grant Program for CDKL5 Deficiency Disorder. CDKL5 deficiency disorder (CDD)…
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