Comprehensive, easy-to-understand information about this condition
How we create this content →Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Jan 30, 2026
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Jan 30, 2026
The documentation for fragile X syndrome type 1 is limited primarily due to its rarity and the absence of systematic clinical studies. Because this condition affects a small population, comprehensive data collection and characterization have been challenging. Additionally, the lack of identified genetic markers further complicates research efforts and clinical understanding.
To navigate your care effectively, consider consulting a neurologist with expertise in genetic neurological disorders, as they may provide insights into managing symptoms and potential treatments. Additionally, while there are currently no identified patient organizations for fragile X syndrome type 1, resources like the Genetic and Rare Diseases Information Center (GARD) at rarediseases.info.nih.gov can offer valuable information. Participating in clinical trials may also be an option, as they can provide access to cutting-edge therapies and contribute to the understanding of this condition.
There are several orphan drugs designated for fragile X syndrome type 1, including blarcamesine, cannabidiol, and Trofinetide, among others. Additionally, there are currently three active clinical trials exploring potential treatments and interventions for this condition. You can find more information about these trials at ClinicalTrials.gov: https://clinicaltrials.gov/search?cond=fragile%20X%20syndrome%20type%201. This research offers hope for future therapeutic options.
Actionable guidance for navigating care for fragile X syndrome type 1
To navigate your care effectively, consider consulting a neurologist with expertise in genetic neurological disorders, as they may provide insights into managing symptoms and potential treatments. Additionally, while there are currently no identified patient organizations for fragile X syndrome type 1, resources like the Genetic and Rare Diseases Information Center (GARD) at rarediseases.info.nih.gov can offer valuable information. Participating in clinical trials may also be an option, as they can provide access to cutting-edge therapies and contribute to the understanding of this condition.
Consider asking your healthcare providers these condition-specific questions
Helpful links for rare disease information and support
Clinical profile data for this condition is not yet available. Phenotype information may still be loading below.
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Jan 30, 2026
Consider asking your healthcare providers these condition-specific questions
AI-Generated Content: This summary was generated using AI. Content has been fact-checked. Always consult with qualified healthcare providers for medical guidance.
Kisho delivers this disease record via API, including phenotypes (HPO), genes, orphan drug designations, screening status, and PAG mapping, with version history and governance.