The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two…
According to a story from BioSpace, the gene therapy companies Sarepta Therapeutics, Inc. and StrideBio, Inc. have recently announced the completion of a licensing and collaboration agreement that could lead…
According to a story from BioPortfolio, the biopharmaceutical company GW Pharmaceuticals plc recently announced that its medicine Epidyolex has earned recommendation from the UK National Institute for Health and Care…
According to a story from rekordmoot.co.za, the du Plessis family of Moot, South Africa couldn't believe it when two year old Barry du Plessis was diagnosed with Dravet syndrome, a…
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 Biotech 365, the biotechnology company Stoke Therapeutics, Inc., recently announced that the very first patient has enrolled for the company's observational study of Dravet syndrome…
According to a story from Pharmaceutical Technology, the company Stoke Therapeutics experimental drug candidate, known as STK-001, has earned Orphan Drug designation from the US Food and Drug Administration (FDA).…
Cluster Seizures Approximately 1/3 of all epilepsy patients live with uncontrolled seizures. Of these, around 150,000 in the United States alone experience cluster seizures (otherwise known as acute-repetitive seizures, crescendo…
There are over 500 children in Scotland whose parents feel could benefit from a new cannabis-derived drug for seizures called Epidiolex. But recently, Secretary Jeanne Freeman had to tell…
According to a story from Science & Technology Research News, a recent study has identified that area of the brain that is associated with "silent" seizures that can appear in…
A new study published in the journal Cell Reports identifies possible new treatments for absence (silent) seizures in children with Dravet syndrome. Children with Dravet syndrome have developmental delays, cognitive…
Each year the Food and Drug Administration approves dozens of new therapies to fulfill unmet needs for various patient populations. It's just been published that in 2018 they approved a…
According to a story from HudsonAlpha Institute for Biotechnology, a team of researchers affiliated with the organization have identified a genetic mutation that is linked a rare pediatric form of…
According to a story from pm360online.com, the pharmaceutical company Zogenix Inc. recently announced that its experimental drug candidate ZX008, also known as Fintepla®, has performed well in the company's latest…
Epidiolex was the first cannabis-based drug approved in the United States. It was first recommended for FDA approval by an advisory committee in April of 2018. In June of this…
November is Epilepsy Awareness Month! Epilepsy is a central nervous system disorder in which brain activity becomes abnormal, causing seizures or periods of unusual behavior, sensations, and sometimes loss of…
In a recent statement, Scott Gottlieb, the Commissioner of the US Food and Drug Administration, has re-affirmed the organisation’s commitment to modernising drug regulatory pathways. New changes are hoped to…
According to a story from Pharma Voice, Dr. Scott Gottlieb, the current commissioner of the US Food and Drug Administration (FDA) has recently released a statement in regards to efforts…
Two Phase 2 clinical trials called ‘Elektra’ and ‘Arcade’ have been initiated. They will investigate the drug OV935/TAK-935 as a potential treatment for paediatric patients with rare epilepsies. For more…
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…
According to Newswire, the two companies, BioMotiv and Cure Network Dolby Acceleration Partners (CNDAP) partnered up together in order to create small molecules that can potentially treat various neurological disorders, including…
The organisations Takeda Pharmaceutical Company and Ovid Therapeutics are collaborating over the development of the experimental drug TAK-935/OV935 as a possible treatment for several rare epilepsy syndromes. Takeda has just…
According to a story from Reuters, the drug development company Zogenix, Inc. has announced in a recent statement that its investigational drug for the treatment of a rare type of…
Zogenix, Inc has announced the top-line results from a Phase 3 study of the investigational drug ZX008 (low-dose fenfluramine hydrochloride) for the treatment of young patients with Dravet syndrome. The…
According to a story from the Washington Post, the US Food and Drug Administration (FDA) has cleared the approval of Epidiolex®, a solution whose active ingredient is a refined version…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
