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Our community is in a relentless pursuit of therapies!
Non - nutritional replacement therapies for CDG
Translating a discovery in biology to a marketable medical breakthrough is a long drawn, tedious, high-investment, and high-risk process. Drug repurposing / repositioning is nowadays a popular strategy. Not quite the same as traditional drug development techniques, the strategy is productive, affordable and is associated with reduced risk compared to novel molecular entities.
An introductory video by Findacure about drug repurposing, aimed at small rare disease patient groups.
What is drug repositioning?
Drug repositioning (also called drug repositioning, reprofiling or re-tasking) is the process of identifying new therapeutic uses for existing and available drugs and for the ones that failed some clinical trial phases (phase 1 and/or 2) for other diseases.
Findacure’s have teamed up with Healx to offer a webinar: 'Drug Repurposing Explained'.
The idea of repackaging an existing drug for another, perhaps more clinically valuable use is not new. Like Rogaine in the 1980s, Viagra was originally a blood pressure drug, opportunistically repurposed when male patients started reporting predictable, long-lasting erections. Traditionally, these secondary effects have been noticed by chance, giving lucky researchers the opportunity to conduct clinical trials to determine whether a given drug can be safely repositioned to treat a different medical condition. But with the rise of next-generation sequencing technology and the age of the $1000 genome, researchers have recently acquired the tools to make smarter decisions about when, why, and how to repurpose a drug (Source: here).
What patients can drug repositioning help?
All people living with CDG.
How can drug repositioning help people living with CDG?
Drug repositioning can accelerate the development of therapies for CDG by reducing the time and costs of the drug development process. It is currently being tested for PMM2-CDG and EXT1/EXT2-CDG. It is also being explored for SRD5A3-CDG.
Below you can see a simplified image about methodologies and steps involved in drug repositioning (Source: Drug Repurposing (DR): An Emerging Approach in Drug Discovery).
Watch the following video made available by aPODD foundation together with Healx, who are researching such potential drug repurposing treatments for an aggressive childhood brain cancer, Medulloblastoma. It helps you understand that drug repurposing offers a potentially faster route to find better treatments for diseases.
Learn more watching below the EJRPD Webinar 1 on Introduction to drug repurposing for rare diseases: benefits, process & patient perspective, co-organised by EATRIS, Radboudumc, FONDAZIONE TELETHON, Les défis sportifs de la Fondation Maladies Rares as part of the European Joint Programme for Rare Diseases (EJP RD).
Keep in mind, you are not alone. Though Congenital Disorders of Glycosylation (CDG) are categorized as “rare” there is an amazing community at your fingertips working day and night to improve the lives of many people living with CDG and their family members. We want you to know everything that is available about therapies in lay language, but we suggest you enter into it at your own pace and comfort level.
Vanessa Ferreira and Sandra Brasil (CDG & Allies FCT, NOVA University, World CDG Organization and Portuguese Association for CDG). Ines Santos, Tiago Martins, Madalena Raposo from Sci and Volunteer Program Nova School of Science and Technology 2021. Ana Sofia Rodrigues (content management, CDG & Allies FCT, NOVA University, World CDG Organization and Portuguese Association for CDG).
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