Pioneering Familial Dysautonomia Treatment: Transforming Lives with BGP-15
Urgency for
Familial Dysautonomia (FD) Solutions
Familial Dysautonomia (FD) presents a critical challenge in rare genetic disorders. While symptomatic treatments have been the mainstay, the quest for disease-modifying therapies is propelled by the urgent need for more effective interventions.
vanguard of this therapeutic revolution
Revolutionizing
FD Care with BGP-15
BGP-15 stands at the vanguard of this therapeutic revolution, offering hope through its targeted approach to mitochondrial dysfunction—a cornerstone in the pathophysiology of FD.
Grounded in rigorous science, BGP-15 has demonstrated its potential in foundational preclinical studies published in the esteemed Proceedings of the National Academy of Sciences (PNAS, Ohlen et al., 2017), laying the groundwork for clinical application.
new milestones
Milestone Achievements in
BGP-15 Development
Marking significant strides, BGP-15 has been conferred Orphan Drug Designation and Rare Pediatric Disease Designation by the U.S. Food and Drug Administration (FDA). These designations underscore the compound’s importance and potential in addressing unmet medical needs within the FD community.
clinical trials
Pathway
to Clinical Impact
The journey from discovery to treatment is marked by a pivotal phase 2/3 clinical trial, set to illuminate BGP-15’s capacity to alter the progression of FD. This trial, approved by the Ministry of Health of Israel, represents a decisive step toward bringing BGP-15’s therapeutic promise to fruition.
safety first
Clinical Prospects and Commitment
to Safety
With a safety profile established in earlier phases, BGP-15 is advancing toward clinical deployment with a focus on efficacy and patient well-being. Its neuroprotective effects hold the promise of not only managing symptoms but slowing the progression of FD.
critical need
Neuronal Preservation
and BGP-15’s Role
BGP-15 extends its influence to the very crux of FD’s impact—the survival of neurons. By rectifying mitochondrial disturbances and enhancing cytoskeletal dynamics, BGP-15 is poised to support neuronal functionality, a critical need for patients with FD.
new chapter in FD care
From Bench to Bedside:
BGP-15's Therapeutic Journey
The trajectory of BGP-15, from preclinical validation to pending clinical trials, is a testament to our commitment to translating scientific discoveries into life-changing treatments. With each milestone, we edge closer
to a new chapter in FD care.
