At #EPNS2025, we are excited to share significant progress in our efforts to address GRIN-NDD. We look forward to engaging with the pediatric #neurology community to collaborate and share knowledge as we continue our development work toward meaningful treatments.

See the program:

We’re honored to join the GRIN community at @CureGRIN’s #GRICON2025, an event that brings together families, researchers, clinicians, and advocates to collaborate and drive progress. Our team is proud to contribute to the program in several meaningful ways.

The @AmerAcadPeds now recommends genome/exome sequencing plus microarray as first-line tests for most children with GDD or ID, expanding support for access to advanced diagnostics that can bring families closer to answers for rare disorders like GRIN-NDD:

We’re proud to sponsor a session at the#2025TSCIRC, hosted by the @tscalliance! Join us in Bethesda, MD as we connect with the community and advance research in tuberous sclerosis complex (TSC). Visit our booth to learn more.

#DidYouKnow that FCD Type II is a rare disorder characterized by malformations of cortical development caused by abnormal brain formation in utero? Learn how we are advancing the Astroscape study for the potential treatment of FCD Type II:

In rare disease research, eligibility criteria must be precise enough to support meaningful outcomes, yet broad enough to reflect the community a therapy is intended to serve. Read our Q&A with SVP Megan Weaver on how we are tackling this delicate balance:

In this @AuthorityMgzine article, Maureen Bennett shares her journey as a mother raising her daughter Kaitlin, who has GRIN1-NDD. Maureen reflects on their family’s path from Kaitlin’s diagnosis to the daily challenges they face:

See our press release:

We are proud to enter into a collaboration with @AngeliniPharma for the development and ex-North American commercial rights of our investigational drug, currently being studied in multiple rare genetic epilepsies and neurodevelopmental disorders.

On #ClinicalTrialsDay, we join the @ACRPDC in celebrating the vital role clinical trials play in advancing research, enhancing our understanding of diseases, and ultimately bringing life-changing therapies to patients. #CTD2025 #PoweredbyPurpose

On #TSCGlobalDay, we join the Tuberous Sclerosis Complex International (TSCi), @tscalliance and @UKTSA to raise awareness of #TSC, a rare genetic disorder caused by variants in the TSC genes that can result in seizures and other neurodevelopmental symptoms.

NMDA receptors are essential for brain function, playing a key role in processes like learning and memory. In individuals with GRIN-related neurodevelopmental disorder, these receptors are abnormal. Learn more: #BehindtheScience

In #raredisease, patient-focused trials ensure research stays centered on what matters most to the communities it serves. As we plan for late-stage development, insights from patients, families, and advocates are shaping our approach. Read more:

We are proud to announce that the European Medicines Agency (EMA) has adopted a positive opinion for the orphan designation for our investigational drug, radiprodil, for the potential treatment of GRIN-related neurodevelopmental disorder. Learn more:

In this @GlobalGenes RareCast podcast episode, our CEO, Bruce Leuchter, M.D., discusses the development of our investigational therapy, radiprodil, for GRIN-related neurodevelopmental disorder (GRIN-NDD). Listen here:

We are thrilled to announce that we have been selected as a finalist for the @Citeline Awards 2025 Excellence in Rare Disease Drug Development. This reflects our commitment to developing targeted therapies for rare neurodevelopmental disorders. Learn more:

We are deeply committed to maintaining strong, ongoing relationships with the patient community, ensuring that their voices remain central to every step of our efforts. Learn more about our partnership with the patient advocacy community:

Learn how we are leveraging our deep understanding of NMDA biology to advance development of a targeted approach to the potential treatment of GRIN-related neurodevelopmental disorder GoF variants:

The ability to modulate the activity of NMDA receptors holds significant potential in treating GRIN-related neurodevelopmental disorder (NDD), as well as tuberous sclerosis complex (#TSC) and focal cortical dysplasia (#FCD). Learn more: