It can be undoubtedly difficult to identify therapies for rare diseases. Between the cost of research, the small population sizes, and the time required, the drug development process can…
The first few years of Saylor Baysden’s life involved a good deal of medical confusion. Her family pursued testing, leading to two diagnoses in 2020-21: autism and epilepsy. But her…
The number of identified rare diseases continues to climb every year, with the number recently reaching the 11,000+ mark. Some of them have only a handful of identified cases, and…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Stoke Therapeutics and Acadia Pharmaceuticals made a joint announcement this week through Business Wire of their collaboration in the development of RNA-based medicines to potentially treat rare neurodevelopmental disorders of…
According to a recent article from BioSpace, Boston’s Children Hospital was granted $308,000 to advance their multidisciplinary biomarker work. SYNGAP1 Syndrome SYNGAP1 syndrome is a neurological disorder that is characterized…
On September 2, 2021, Patient Worthy attended a webinar from the SYNGAP Research Fund titled "Treatments in Development for Epilepsy Syndromes: Opportunities for SYNGAP1." This program provided an overview of…
When Caren was eighteen years old her parents were, in fact, given two options. If they were unwilling to consent to a lobotomy, Caren would remain in the dark ward…
In a recent press release, the SynGAP Research Fund (SRF) shared that the Heller Laboratory of Neuroepigenetics would receive a $130,000 research grant. The fund, which will be granted over…
A concerned Texas mom finally has answers about the cause of her son’s sensory-processing problems, thanks to a special collaboration among her patient-advocacy group and the lab of Scripps Research…
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