Orsini - Rare Disease Pharmacy Solutions

Today, we celebrate Rare Disease Day! This week, we gathered as a team to hear inspiring stories that touched our hearts, granted a wish trip for a family living with spinal muscular atrophy, and honored the courage and resilience of the rare disease community we are so proud to serve. And we had a lot of fun along the way...can you spot who greeted our team in a zebra suit Monday morning? ?? Together, we are committed to making a difference and raising awareness for rare diseases. ?? Happy Rare Disease Day! ?? #RareDiseaseDay #ShowYourStripes #NoPatientLeftBehind

Meet Angelo, a remarkable 8-year-old boy who’s facing the challenges of spinal muscular atrophy (SMA), a rare neuromuscular disease, with incredible courage. Despite using a wheelchair and having physical limitations, Angelo’s positivity is contagious. One of his favorite things to do is swim – because in the water, he can move freely and feel limitless. Like many boys his age, Angelo loves video games and movies. It was through watching the Sonic movie that Angelo first discovered Hawaii – the beautiful landscapes and the ocean captured his heart. Since then, it's been his dream to visit Hawaii and swim in the ocean with his siblings. In honor of Rare Disease Day, we are incredibly excited to partner with the Make-A-Wish Greater Bay Area to make wishes like Angelo’s come true. Thanks to our support, Angelo and his family will enjoy a life-changing wish trip to Hawaii. Please join us in celebrating Angelo’s courage and resilience, and in supporting the wonderful work of Make-A-Wish. ??? #RareDiseaseDay #MakeAWish #SMA #NoPatientLeftBehind

"When Sawyer was just 18 days old, he received gene therapy treatment that saved his life. I can't fully capture the overwhelming emotions of watching that tiny vial—the symbol of hope we'd been holding onto—finally infused into our son. It was a moment that stirred a whirlwind of emotion—relief, fear, gratitude, and a quiet sense of uncertainty. It felt like we were crossing a threshold, unsure of what would come next but hopeful and confident that this was the best decision for Sawyer." -Francesca Hansen, mother of Sawyer This week, we were honored to hear a powerful story from the family of a patient, whose journey with gene therapy is a testament to hope and courage. Thank you to the Hansen family for sharing your inspiring story with our team in honor and celebration of Rare Disease Day. ?? #RareDiseaseDay #GeneTherapy #Inspiration #NoPatientLeftBehind Learn more about how we met the Hansen family in this post: https://lnkd.in/eJ-m56aR

Rare Disease Day…what does it mean to you? As we raise awareness throughout February leading up to?#RareDiseaseDay?on February 28, we stand with the rare disease community, committed to ensuring every patient receives the care, support, and access they deserve. Swipe through to see what inspires our team to do this important work. What does Rare Disease Day mean to YOU? Share in the comments! #NoPatientLeftBehind?#RareDiseaseAwareness?#CompassionateCare

On this first Monday of February, we’re kicking off a month of awareness leading up to #RareDiseaseDay on February 28. Two-thirds of Americans with rare diseases are children, many diagnosed at birth or early childhood, and these families face unique challenges navigating care. At Orsini, we understand these challenges and have designed our care model to provide specialized support and compassionate care for young patients and their families. ?? Watch this video to see how our high-touch care model is tailored to meet the needs of children with rare diseases, ensuring no patient is left behind: https://lnkd.in/eUZgehUt Let’s continue to shine a light on rare diseases and work together to improve the lives of patients and families living with a rare condition. #RareDiseaseDay #NoPatientLeftBehind #RareDiseaseCare #CompassionateCare

Did you know that Orsini offers hub services, integrated with our specialty pharmacy workflow to create a seamless experience for both our patients and our biopharma manufacturers? Find out more on our website: https://lnkd.in/g-Z9wEB5 #NoPatientLeftBehind #PatientCare #PatientServices

Today, we honor the life and legacy of Dr. Martin Luther King Jr. His unwavering dedication to justice, equality, and compassion continues to inspire us as we serve rare disease patients and their families. At Orsini, we are committed to carrying forward his vision by ensuring every patient has access to the care they need, regardless of the challenges they face. Let this day serve as a reminder for us all to work together toward a brighter, more equitable future. #MLKDay #OrsiniCares

Orsini is proud to be the exclusive specialty pharmacy partner selected by PTC Therapeutics, Inc. for KEBILIDI (eladocagene exuparvovec-tneq), a gene therapy for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. “As the leading independent specialty pharmacy in cell and gene therapies, Orsini is well-positioned to help those suffering from AADC deficiency gain access to KEBILIDI,” Brandon Tom, Orsini’s CEO, said. “We are always honored to be entrusted with the care of those affected by rare diseases, particularly pediatric patients and their families, and we look forward to partnering with PTC to offer new hope to the AADC community.” Read the press release: https://bit.ly/4h5pp6q #AADC #CAGT #CGT #PatientCare #RareDisease #NoPatientLeftBehind

In celebration of National Pharmacist Day this Sunday, we’re shining a spotlight on the heart and soul of our pharmacy team—our pharmacists. Hear directly from them about what makes their work so rewarding and why they're passionate about making a difference in the lives of others every day. #NationalPharmacistDay #PharmacyHeroes #NoPatientLeftBehind

Our team is eager to connect with clinicians, researchers, and industry partners who share our passion for improving rare disease patient care at Parent Project Muscular Dystrophy's #Duchenne Healthcare Professionals Summit next week. We hope to see you there! #PPMD #RareDisease #PatientCare #EndDuchenne