Dr. Paulina Rychenkova

accelerating path to treatments for rare neurodevelopmental disorders @CureSHANK; partnering with innovative companies @AbbVie

[CureSHANK @CureSHANK]

We're joining forces w/ @Phelan_McDermid for #PMSAD 10/22!

We’ve got free resources to raise awareness & share your journey:

🌐 Get it all here: https://t.co/koiAANCk1Q.

Be sure to tag us in your posts & include hashtags #PMSAD & #EveryStoryShinesGreen!

[Ana Mingorance @CNSdrughunter]

🚨 Attention biotech research and investors: we are launching the @CureSHANK Therapeutic Accelerator and hosting an Open House at #JPM25 #JPM2025

Phelan-McDermid syndrome (SHANK3 haplo) is a NDD/autism/DEE syndrome with 1 in 15,000 incidence. We want to accelerate treatments🚀

[Melinda French Gates @melindagates]

This is a discouraging day for so many people across our country and around the world. But make no mistake: Nothing ends here.

While many of us hoped for a different outcome, we knew that this was a possibility. Advocates, activists, and champions for Americans of all backgrounds and identities have prepared for this moment, and they are ready to work at all levels using every tool at their disposal to ensure that progress keeps moving forward.

Over the next four years, we will see heroic work across our country—in our state legislatures, in our court system, in community organizations distributing frontline services, and everywhere in between. I am committed to being part of those efforts and know that I will be just one of tens of millions of people who are ready to work together to prove that no matter who holds the office of the president, the United States will continue its march to a more equal future.

[Ana Mingorance @CNSdrughunter]

@Phelan_McDermid @CureSHANK Block 3 at the #PMSDDS2024 is about critical questions in clinical trials and what we can learn from other diseases to help therapy development for #SHANK3

[Ana Mingorance @CNSdrughunter]

@Phelan_McDermid If everything goes well, #SHANK3 will soon have its first pivotal trial AND its first gene therapy trial. We review the data and plans from Neuren and Jaguar at the #PMSDDS2024

[Ana Mingorance @CNSdrughunter]

The #PMSDDS2024 meeting just started, hosted by @CureSHANK, reviewing the state of the art and path to precision medicines for #SHANK3 / Phelan-McDermid 🧠🦓🧬

[Ana Mingorance @CNSdrughunter]

🧠 Neuroscientist in the Boston area?
🦓 or attending the #WODC orphan meeting in April?

Then check out the #SHANK3 Phelan-McDermid symposium that @CureSHANK is hosting at @MassBio Hub on April 26 right after the WODC.

Details, program and registration: https://t.co/hvReKPYtVJ

[CureSHANK @CureSHANK]

Neuren Pharmaceuticals just announced positive top-line results from its Phase 2 Trial of NNZ-2591 in #PhelanMcDermidsyndrome. CureSHANK is a proud supporter of this endeavor, and we look forward to helping Neuren to move quickly to its Phase 3 trial.
https://t.co/lNecGg0y6q

[CureSHANK @CureSHANK]

We are excited to join @MilkenInstitute's @fastercures #FCTRAIN, a program for innovative nonprofits funding medical research. TRAIN organizations are committed to accountability, collaboration, research, resources, and patient-centeredness. https://t.co/AQ9XL5NvVZ

[CureSHANK @CureSHANK]

CureSHANK is excited to announce a request for applications (RFA) for our $250,000 CureSHANK Research-to-Cure Grant: EPILEPSY. This grant aims to stimulate research on SHANK3-related epilepsy.

View the RFA here: https://t.co/tVvEI1SRmZ

[Dr. Paulina Rychenkova @paulinarych]

@CNSdrughunter @aloktayi What can advocacy groups like @CureSHANK do to help address these concerns with the FDA?

[Dr. Paulina Rychenkova @paulinarych]

@CNSdrughunter @aloktayi That is hugely discouraging to all rare disease patients and caregivers. Also inconsistent with the rhetoric rare disease groups have been hearing from the FDA over the past several year.

[CureSHANK @CureSHANK]

Be sure to *pre-register* for @CureSHANK's Fall 2023 Community Webinar!
Join us to learn about CureSHANK's groundbreaking work this year; what is in the works; and how YOU can help advocate for treatments for #phelanmcdermidsyndrome!
Pre-register at: https://t.co/bzhbGdauky

[Dr. Paulina Rychenkova @paulinarych]

Privileged to speak at 2nd European #phelanmcdermid syndrome summit and represent @CureSHANK Great energy thanks to @PhelanMcDermid

[Asociación Síndrome de Phelan McDermid España @PhelanMcDermid]

REMEMBER! 

If you sign up for the streaming of the 2nd European Conference on #PhelanMcDemidSyndrome (only for non residents in Spain), you will have access to all the presentations for a month, so you can watch them when it suits you best. 

Sign up! https://t.co/YCxiSAJDDt

[Fyodor Urnov @UrnovFyodor]

Stunning-size audience at the American Society of Cell and Gene Therapy: 8,000 folks working on genomic therapy cures. #ASGCT2023
A goal for us all:
8,000 persons treated with CRISPR for N=1/rare genetic disease by ASGCT 2033.
This is a moral must: >100 million patients await.

[CureSHANK @CureSHANK]

We did it! Special thanks to @Talya for her tireless efforts which translated into us raising $90,000+ in five days!

#phelanmcdermidsyndrome #CureSHANK #epilepsy #developmentalregression #sleepdysfunction #autism #neurodevelopmentaldisorder #anythingforasa

[CureSHANK @CureSHANK]

Help us figure out better treatments—better yet, a cure!—for #epilepsy in #phelanmcdermidsyndrome.

We are #raisingforresearch May 1-5 at https://t.co/npcop8okr1 .

[Dr. Paulina Rychenkova @paulinarych]

Day 4: another opportunity to support research into core debilitating conditions associated with #phelanmcdermid syndrome.

[CureSHANK @CureSHANK]

https://t.co/ZvrgLb1vla

[CureSHANK @CureSHANK]

Help us figure out better treatments—better yet, a cure!—for #epilepsy in #phelanmcdermidsyndrome.

We are #raisingforresearch May 1-5 at https://t.co/npcop8okr1 .

https://t.co/YbdIJZynP8