PTC Collaborates with RareMed to Enhance PKU Patient Support with Sephience™ Therapy
- RareMed Solutions expands collaboration with PTC Therapeutics to enhance support for PKU treatment via Sephience™.
- The partnership increases dedicated liaisons for personalized care and improved patient experiences in managing PKU.
- This collaboration emphasizes the importance of patient-centric care as new therapies for PKU emerge.

New Collaboration Enhances Patient Support for PKU Treatment
RareMed Solutions® recently announces an expanded collaboration with PTC Therapeutics™ to provide non-commercial pharmacy dispensing services for Sephience™, a therapy recently approved by the FDA for the treatment of phenylketonuria (PKU). This partnership is poised to make a significant impact on the lives of both adult and pediatric patients suffering from this rare, inherited metabolic disorder. PKU results from a genetic defect that prevents the body from producing an enzyme essential for breaking down phenylalanine, an amino acid that can accumulate to dangerous levels if left unmanaged. The introduction of Sephience™ represents a crucial advancement in treatment options for the approximately 58,000 individuals affected worldwide.
The collaboration between RareMed and PTC Therapeutics aims to enhance patient support through an expanded RareSupport® Team, which will increase the number of liaisons dedicated to assisting patients and healthcare providers. This initiative is designed to facilitate comprehensive outreach and coordination, ensuring that patients receive personalized care tailored to their unique treatment journeys. With a focus on compassion and accessibility, RareMed’s framework is set to provide essential guidance and timely assistance, which can significantly improve the overall treatment experience for all stakeholders involved. The growing complexity of managing PKU underscores the importance of such support services, particularly as new therapies emerge.
RareMed's commitment to enhancing patient experiences is reflected in its strategic growth, which positions the company as a leading concierge patient services provider for complex conditions. This dedication to adapting to the evolving needs of patients not only reinforces RareMed's reputation but also exemplifies the critical role that patient support services play in the broader healthcare landscape. As more therapies become available, the need for effective coordination and patient education becomes increasingly important, and RareMed’s efforts to expand its services are timely and necessary.
In addition to the launch of Sephience™, RareMed's enhanced support services aim to address the multifaceted challenges that PKU patients face. By providing dedicated liaisons, the company ensures that patients can navigate their treatment pathways with greater confidence and understanding. This approach not only helps mitigate the risks associated with untreated PKU but also fosters a supportive community for patients and their families.
Overall, this collaboration between RareMed and PTC Therapeutics marks a vital step forward in the fight against PKU, showcasing the importance of patient-centric care in the management of complex therapies. As the healthcare sector continues to evolve, partnerships like this one highlight the necessity of tailored support services that prioritize patient outcomes and quality of life.