Proud to share the first video of the yeasts screenings being conducted in the Andrew Munkacsi of Te Herenga Waka—Wellington Uni Research with Ethan Perlstein bio/acc and our Perlara Cure Guides in the search for the #GNAO1 #G203R treatment. vimeo.com/791862350 #DrugMultipurposing #PerlaraPioneerFamilies

Proud of our Perlara team on the first major milestone in finding the first treatment for a variant of the #GNAO1 mutation.

Data hot off the presses! We performed a screen to assess the activation of a GPCR signaling pathway in a GNAO1/GPA1 yeast avatar with reduced pathway activity at baseline. Hit 1 (left) versus untreated control (right)

Our history so far.... #DrugMultipurposing #PerlaraPioneerFamilies

Our first image-based yeast cell phenotypic screen yielded drug multipurposing hits! As we’ve seen time and time again, the hits are unique to this gene/disease.

Patients are the true limiting reagent in the crucible of drug development. It's time we recognize this reality, and build a care-to-cure system that rewards patients with both cures and cash. perlara.substack.com/p/patients-are…

We’re simply not making progress fast enough. We need 100s of new medicines approved per year to even begin to make a dent in all 9,000+ rare diseases. It feels good to commune with others in person or on social media today, but good feels won’t make medicines.

On Rare Disease Day, this ir our message today. #RareDiseaseDay2023

It just takes one family and one physician to try a drug, discover novel biomarkers, share all the data, and make enormous impact. The reality is to get started on your cure odyssey you don’t need a registry or a natural history study — or even a community for that matter.

Unlocking the secrets of the #G203R variant #GNAO1 Doing science and getting closer to treatment.

In partnership with GNAO1 Action, G203R yeast avatar drug repurposing screens are done 💊♻️ ✅ Next stop: 1-to-N pioneer studies perlara.substack.com/p/gnao1-g203r-…

Welcome to Europe!

Proud of the great achievement! We move forward, the BEST is yet to come💫💫💫

Hoy 1 de octubre conmemoramos el día de concienciación sobre las mutaciones en el gen GNAO1. ¿Y si pasamos a la acción? La cura está más cerca que nunca y la esperanza para tantos niños que están y que estarán. Esa es la mejor celebración que podemos hacer en este día. #GNAO1

Pediatric clinicians: Do you have patients with at least one childhood-onset movement disorder and any of these genes? Become a contributor for this retrospective multicenter study with just a short data submission: loom.ly/928aVu0

Want to see what a direct-to-patient biotech pipeline looks like? 22 programs funded by pioneer families, 12 in various stage of 1-to-N clinical studies, and half of hits are clinically actionable nutraceuticals. This is how you start to do drug repurposing at scale.

Millones de gracias Pedro Moreno Gómez. 💚🇪🇸 por la implicación con las enfermedades raras y la discapacidad. 🩵🩵🩵

Closer and closer to treatment for our children! We continue to move forward on the right path. Proud of everything we have achieved so far with our friends from Perlara, together on the path of BioAcceleration. #GNAO1 #G203R

Following the same path