Ganaxolone Achieves Primary Endpoint in Phase 3 Trial for CDKL5 Deficiency Disorder (CDD), a Rare Form of Genetic Epilepsy22 Sep 2020
Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced positive top-line results from its registrational Phase 3 clinical trial (Marigold Study) evaluating the use of oral ganaxolone in children and young adults with CDKL5 deficiency disorder (CDD), a rare, genetic epilepsy with refractory seizures.Read More >>
Marinus Pharmaceuticals Receives Rare Pediatric Disease Designation from FDA for Ganaxolone for the Treatment of CDKL5 Deficiency Disorder (CDD)30 July 2020
Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, announced today that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation for the company’s product candidate ganaxolone for the treatment of CDKL5 deficiency disorder (CDD).Read More >>
CDKL5 Deficiency Disorder Therapeutic Pipeline and Community Update - July 202011 July 2020
Antonino Caridi, President of the CDKL5 Alliance, and Majid Jafar, co-Founder of the Loulou Foundation, reviewed the therapies in development for CDKL5 Deficiency Disorder. The video starts with a short update of the pipeline followed by a discussion between Antonino and Majid where they addressed the questions from the audience.
CDKL5 Deficiency Disorder Voice of the Patient Report Submitted to FDA18 June 2020
Understanding the patient voice is fundamental for therapy development. Companies need to ensure that what matters most to patients is being assessed, and regulators and payers asks for demonstration of value to support approval and reimbursement. Today, the Loulou Foundation and the International Foundation for CDKL5 Research (IFCR) are proud to release the “CDKL5 Deficiency Disorder Voice of the Patient Report”, capturing the voice of patients living with this rare genetic disease that affects thousands of people.Read More >>
Main lessons from the 2019 CDKL5 Forum13 November 2019
The 5th edition of the CDKL5 Forum recently took place in Boston, in November 4 and 5. The Forum is an annual meeting hosted by the Loulou Foundation where scientists and drug developers working on CDKL5 deficiency, together with representatives from patient organizations, meet to discuss the latest advances. This was the third Forum I attended, and the second since joining the Loulou Foundation.
Because of the 5th anniversary, the CDKL5 Forum Director (and my dear friend) Dan Lavery offered an update of how much the entire CDKL5 deficiency field has changed since the first Forum edition. And it has changed so much that it seems we are looking at two different diseases!Read More >>
Main lessons from the 2018 CDKL5 Forum25 October 2018
For the past four years the Loulou Foundation hosts an annual “by invitation only” meeting where scientists and drug developers working on CDKL5 deficiency, together with representatives from patient organizations, meet to discuss the latest advances. This was the second Forum I attended, and my first since joining the Loulou Foundation.
There were major news announced during the meeting, like a new Phase 2 clinical trial in CDKL5 deficiency disorder with fenfluramine, a drug that has completed two Phase 3 clinical trials in Dravet syndrome with very strong efficacy, and the announcement that Ultragenyx will develop a gene therapy for CDKL5 deficiency disorder...
Ovid Therapeutics Receives “Company Making a Difference Award” from CDKL5 Deficiency Disorder Community24 October 2018
Ovid Therapeutics Inc., a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, today announced it has received the 2018 CDKL5 ForumCompany Making a Difference Award for initiation of the Phase 2 ARCADE trial with OV935/TAK-935, and its commitment to the CDKL5 deficiency disorder (CDD) patient community. The award was announced today in London, UK at the CDKL5 Forum, the largest annual conference on the advancement of science and therapeutic development for CDD...Read More >>
Ultragenyx and RegenXBio Announcement for Gene Therapy to tackle CDKL5 Deficiency Disorder22 October 2018
Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced it has exercised its option with REGENXBIO to develop a gene therapy to treat patients with CDD (CDKL5 Deficiency Disorder) utilizing REGENXBIO’s adeno-associated virus (AAV) vectors, including AAV9.Read More on Ultragenyx >> Read More on Regenxbio >>
Amicus Therapeutics Enters Research and Development Collaboration with University of Pennsylvania to Develop AAV Gene Therapies8 October 2018
Amicus Therapeutics today announced a major collaboration with the Gene Therapy Program in the Perelman School of Medicine at the University of Pennsylvania (Penn) to pursue research and development of novel gene therapies for Pompe disease, Fabry disease, CDKL5 deficiency and one additional undisclosed rare metabolic disorder. This relationship will combine Amicus’ protein engineering and glycobiology expertise with Penn’s adeno associated virus (AAV)...Read More >>
Ovid Therapeutics Announces Initiation of Two Phase 2 Clinical Trials of OV935/TAK-935 for Pediatric Patients with Rare Epilepsies24 September 2018
Ovid Therapeutics Inc., a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, today announced initiations of the Phase 2 ELEKTRA and ARCADE trials for OV935/TAK-935 in pediatric patients with rare epilepsies. The company reports that the first patient has been randomized to receive either OV935 or placebo in the ELEKTRA trial and that patient screening is underway for the ARCADE trial...Read More >>
Marinus Pharmaceuticals Initiates Pivotal Phase 3 Trial in Children with CDKL5 Deficiency Disorder19 June 2018
Marinus Pharmaceuticals, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and neuropsychiatric disorders, today announced the initiation of a pivotal Phase 3 clinical trial (Marigold Study) evaluating the use of oral ganaxolone in children and young adults with CDKL5 Deficiency Disorder (CDD)...Read More >>