October is Rett Syndrome Awareness Month.
Rett syndrome is a neurodevelopmental condition that primarily affects girls. People with the disease appear to have normal psychomotor development during the first 6 to 18 months of life before developing severe problems with language and communication, learning, coordination, and other brain functions. Additional signs and symptoms may include repetitive, stereotypic hand movements, fits of screaming and inconsolable crying, autistic features, panic-like attacks, teeth grinding, episodic apnea and/or hyperpnea, gait ataxia and apraxia, tremors, seizures, and slowed head growth. Classic Rett syndrome is most commonly caused by genetic changes in the MECP2 gene.
The Rett syndrome landscape is always evolving, including better diagnostic understanding and novel treatment options that are improving patients outcomes. Notably, Daybue (trofinetide) was approved by the US Food and Drug Administration (FDA) in March 2023, and in December 2025 was granted expanded approval to a new formulation, Daybue Stix, as a powder that can be dissolved in a flavored liquid for ease of use. Such advancements are making way for patient-centered approaches to rare disease care.
Rett Syndrome Awareness Month is a time dedicated to increasing understanding of Rett syndrome and supporting the individuals and families impacted by this rare neurological condition. It is also an opportunity to recognize the importance of continued research, multidisciplinary care, patient advocacy, and education within the rare disease community.
At CheckRare, we are committed to raising awareness of rare conditions like Rett syndrome and helping connect healthcare professionals and patients with timely educational resources, expert insights, and emerging clinical developments.
To learn more about Daybue Stix, visit https://checkrare.com/daybue-trofinetide-stix-new-formulation-of-treatment-for-rett-syndrome/
For more information on real-world data from the LOTUS clinical trial of Daybue in patients with atypical Rett syndrome, visit https://checkrare.com/lotus-study-in-patients-with-rett-syndrome-treated-with-daybue-trofinetide/
Rett Syndrome Advocacy Groups:
International Rett Syndrome Foundation