The Gift of Anticipatory Guidance: What Every Family Deserves
- September 10, 2025
A Decade of Progress: Gene Therapy Delivers Lasting Relief for Hemophilia B
A New Era for Fragile Skin: How Gene Therapy is Transforming Rare Skin Diseases
Understanding the Patient Experience with Twice-Nightly Sodium Oxybate Therapy for Narcolepsy: A Social Listening Experiment
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@SyngapResearchFund #RareDisease #SYNGAP1"Emmy faces a range of challenges: seizures, autism, hypotonia, cortical visual impairment, high pain tolerance, sleep disturbances, and elopement. She requires constant vigilance. At the same time, Emmy fills our days with joy."
#WarriorWednesday 🧬 Emmy's full story here 👉 cureSYNGAP1.org/Warrior
Emmy's parents are hosting the Beacon of Hope, A Benefit For 𝘚𝘠𝘕𝘎𝘈𝘗𝟣 on 9/12/25 in Boston - help us #CureSYNGAP1 by donating at cureSYNGAP1.org/Beacon25
#SRFWW no. 223
#SYNGAP1 💙 💜 💚
September is STXBP1 Awareness Month! Join us in spreading hope, knowledge, and support for those affected by STXBP1-related disorders. Every story matters and every voice counts! #stxbp1awarenessmonth #hopeforstxbp1 #RareDisease #shareyourstory #PatientWorthy
If you're interested in sharing your story, click here: bit.ly/4dV7gru
Thank you Tom Seaman! #Dystonia #dystoniaawareness
