Harmony Biosciences

At Harmony Biosciences, we believe that when empathy and innovation meet, a better future can begin for people living with rare neurological diseases, their families and communities. In 2023, we expanded our commitment to the Fragile X syndrome community by acquiring Zynerba Pharmaceuticals. We are currently conducting the RECONNECT Phase 3 clinical trial, designed to confirm the positive findings from the phase 2/3 CONNECT study. If successful, this has the potential to be the first and only approved treatment for patients with Fragile X syndrome.

Shionogi

Shionogi Group Company is focused on developing treatments for central nervous system diseases and disorders with a lead program in Fragile X syndrome (FXS). Shionogi acquired Tetra in 2020 as part of its mission to develop medications for unmet medical needs and to contribute meaningfully to society. Shionogi and Tetra are researching an investigational treatment called zatolmilast for FXS. The U.S. Food & Drug Administration has granted zatolmilast Orphan Drug Designation, Rare Pediatric Disease Designation and Fast Track Designation. If approved, zatolmilast could offer people living with FXS the first cognitive treatment developed specifically for them. Learn about their research and find out more about the eligibility criteria to join their study here.

Mirum Pharmaceuticals

Mirum Pharmaceuticals is a multi-national biopharmaceutical company focused on developing medicines for children and adults with rare diseases. At Mirum, our need to develop transformative medicine is propelled by our passion for patients and their loved ones, and we’re focused on turning possibilities into realities. In 2024, Mirum acquired MRM-3379 as part of its mission to help fill therapeutic voids in underserved and undertreated diseases. Mirum is planning a Phase 2 study to evaluate MRM-3379, an investigational, phosphodiesterase- 4D (PDE4D) allosteric inhibitor that in research could offer a novel approach in the treatment of Fragile X syndrome (FXS) by assessing the potential of MRM-3379 to block PDE4D, thereby potentially restoring transcription of genes involved in synaptic plasticity to help improve cognition, language, and daily function. For more information about MRM-3379, click here.

Developmental FX

Developmental FX (DFX) is a thought leader in the realm of pediatric occupational therapy, multidisciplinary pediatric care, clinical reasoning, sensory integration, and trauma-informed care. We are developing and sharing models of care and best practices through research, publications, resources, teaching, mentoring, podcasts, and presentations delivered online and in person.

In our state-of-the-art Denver clinic, we serve over 500 children per year and reach thousands more through programs that go beyond the clinic to share our Therapy that Fits® philosophy with clinicians, educators, caregivers, and other providers. This innovative approach integrates developmental therapeutic practices informed by cutting-edge neuroscience and emphasizes relationship-based interventions that support the whole individual. The client population served includes neurodivergent groups, autism, ADHD, sensory integrative needs, anxiety, learning differences, motor coordination difficulties, and other developmental, learning, mental health, and behavioral differences. We also have a high degree of specialization to address genetic and rare conditions, grounded in our founders’ deep connections to the Fragile X community.

CEO Commission for Disability Employment

The CEO Commission for Disability Employment was founded by Voya Financial, the Society for Human Resource Management (SHRM), and the National Down Syndrome Society (NDSS), to advance disability inclusive employment by inspiring and engaging business leaders to drive change through policy, practice, and culture.