Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
Quinnlyn Walters is a bright, bubbly, and active 8-year-old. She loves reading, riding horses, swimming and softball, karate, Star Wars, and spending time outside with her friends and family. But…
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,…
When Brittany Miller welcomed her daughter Brooke into the world, she had no idea what the next few months would bring. At just a few weeks old, Brooke began having…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
Did you know that June is considered Dravet Syndrome Awareness Month? Every year, people within the Dravet syndrome community come together to amplify stories, raise awareness, and encourage the general…
Prior to the end of March 2023, ganaxolone had received six separate Orphan Drug designations from the FDA; more recently, as shared in a news release from commercial-stage pharmaceutical…
Seizures associated with conditions such as Dravet syndrome and Lennox-Gastaut syndrome (LGS) can be difficult to treat; these seizures may be treatment-averse and may not respond well to current anti-epileptic…
When Erika Vandenberg learned that her daughter Aubrey had a rare genetic disorder called SLC6A1, her first two reactions were fear and relief. Relief because they finally had a name…
At 20 years old, Grace Hinchman was - for all intents and purposes - a happy and healthy young adult. Grace was studying Business at Loyola University Chicago and playing…
According to a recent article, the parents of a boy with epilepsy were told they would need to prepare for him to have end-of-life care after they were unable…
According to a recent announcement, Epilepsy Research UK has announced a funding collaboration with the Tuberous Sclerosis Association which will support the investigation of tuberous sclerosis complex (TSC)-associated epilepsy.…
During this month, the Dravet Syndrome Foundation (DSF) organizes a variety of activities and resources to help raise awareness for Dravet syndrome, a rare form of epilepsy. They make it…
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 the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions. A “rare” condition is one affecting under…
What do medication, vagus nerve stimulation, and low-carb ketogenic diets all have in common? Currently, these are all therapeutic options for those with Dravet syndrome. However, researchers and doctors believe…
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…
A study conducted by Rayyan Zafar and his colleagues from the Imperial College London was recently published in BMJ Paediatrics Open. This investigation evaluated the efficacy of whole-plant cannabis as a treatment for…
When Shelby Sparks was just five months old, she began having grand mal seizures. Throughout her early years, her family just believed that their daughter had severe epilepsy. Then, when…
When you think of effective fundraisers, do you think of pickleball? Well, a Kansas community did just that. According to a video and transcript on KMBC News 9, his community…
According to an August 17, 2021 news release from pharmaceutical company Marinus Pharmaceuticals, Inc. (“Marinus”), data is available from a Phase 2 clinical trial evaluating oral ganaxolone for patients…
It’s no secret that cannabis and its components are highly controversial. However, cannabis has been used to treat a variety of conditions, including seizures. According to Candid Chronicle, another related…
Rachel Ablondi recently wrote an article expressing her journey and thoughts on her son's life with Lennox-Gastaut syndrome (LGS) in an effort to get a message across to doctors. While…
According to a recent news release, the Lennox-Gastaut Syndrome (LGS) Foundation shared that its 6th Annual Walk 'n' Wheel event will take place on June 5th, 2021. The event…
Inbrain Neuroelectronics has announced that they have raised 16.8 million dollars which will support clinical testing of a new device for Parkinson's disease as well as epilepsy. This is a…
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