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 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…
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…
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…
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