RareMed Announces Expansion of Relationship with PTC Therapeutics
PITTSBURGH, Sept. 2, 2025 /PRNewswire/ -- RareMed Solutions®, the nation's leader in patient support services for complex therapies, is proud to announce an expansion of its relationship with PTC Therapeutics(TM) with the launch of non-commercial pharmacy dispensing services for PTC's therapy, Sephience((TM)). The recent approval of Sephience by the FDA marks a significant milestone in the treatment of phenylketonuria (PKU) for both adult and pediatric patients. This new therapy, developed by PTC, has the potential to transform the lives of those living with PKU by offering an innovative treatment option that addresses their unique needs.
Support for Patients and Providers
RareMed will be providing non-commercial pharmacy dispensing services to support patients in challenging circumstances. These services are designed to provide both patients and healthcare providers with appropriate assistance.
To provide seamless access and support, RareMed RareSupport® Team is expanding. A larger team of liaisons will play a crucial role in providing outreach and coordination efforts across PTC's supported therapies so that every patient may receive appropriate, personalized care and attention, while leadership will provide guidance, oversight, and compliance support.
Commitment to Care
RareMed's commitment to providing care extends beyond just the launch of new therapies. The program's support framework is designed to assist patients and providers alike in navigating the treatment landscape with confidence. By expanding the team, RareMed aims to facilitate a smoother, more coordinated experience for all stakeholders involved in the treatment process.
As RareMed continues to grow and adapt to the evolving needs of its patients, the company's focus remains steadfast on delivering compassionate, effective, and timely care.
About Phenylketonuria
Phenylketonuria (PKU) is a rare, inherited metabolic disease, which affects the brain. It is caused by a defect in the gene that helps create the enzyme needed to break down phenylalanine (Phe). If left untreated or poorly managed, Phe--an essential amino acid found in all proteins and most foods--can build up to harmful levels in the body. This causes severe and irreversible disabilities, such as permanent intellectual disability, seizures, delayed development, memory loss, and behavioral and emotional problems. Newborns with PKU initially do not have any symptoms, but symptoms are usually progressive, and damage caused by toxic levels of Phe in the first few years of life is irreversible. Diagnosis of PKU usually takes place during newborn screening programs. There are an estimated 58,000 people living with PKU globally.
About RareMed
RareMed Solutions is the nation's only concierge patient services provider, delivering award-winning care to patients living with complex conditions. RareMed partners with biopharma to transform the lives of patients afflicted with devastating complex conditions by accelerating access to biomedical breakthroughs. RareMed offers case management, co-pay, coupon and financial assistance programs, reimbursement support, nursing support, healthcare professional education, patient adherence & education, and non-commercial pharmacy dispensing services to all fifty states from its headquarters in Pittsburgh, Pennsylvania and bases across the country. RareMed's advanced AI-integrated hub technology offerings, including the revolutionary RarePath® platform, provide unparalleled management and analytics capabilities to biopharma across the nation.
The company has a breadth of experience developing, transitioning, and maintaining therapy-specific solutions that ensure unparalleled manufacturer & patient satisfaction across a variety of patient services models. RareMed's undivided complex disease focus, high caliber associates, fully dedicated teams, and sophisticated proprietary technology enable it to address the unique needs of complex disease patients. RareMed has been consistently recognized as a top workplace in the nation by USA Today and Newsweek and continually raises the benchmark for patient experience according to MMIT patient satisfaction surveys, in which the team recently achieved a perfect 100-point net promoter score. Visit RareMed's website at www.RareMed.com.
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