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PITTSBURGH, April 1, 2024 /PRNewswire/ -- PANTHERx®Rare, a leader in rare disease product patient access and support services in the United States, is pleased to announce that it was selected by Edenbridge Pharmaceuticals for the distribution of Yargesa® (miglustat) capsules, the first oral treatment option for adults with mild to moderate Type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option.

Yargesa® is a glucosylceramide synthase inhibitor that exerts its effects through a process called substrate reduction therapy. Through this process, Yargesa® reduces the buildup of harmful glycosphingolipids and symptoms of the disease.

Gaucher disease is a rare, genetic lysosomal storage disorder where individuals lack the functioning glucocerebrosidase enzyme leading to the accumulation of a certain lipid known as glucosylceramide. This accumulation leads to manifestations including but not limited to enlarged spleen or liver, hepatomegaly, anemia, thrombocytopenia, and bone pain.

"We appreciate Edenbridge Pharmaceuticals' commitment to making a difference in the lives of patients with Type 1 Gaucher disease. We are looking forward to serving the needs of the patients dealing with this illness, by streamlining the process associated with getting rare medications from the people who create them to the people who need them most, ultimately delivering better health outcomes to people living with rare diseases," said Rob Snyder, CEO of PANTHERx® Rare Pharmacy.

About PANTHERx Rare

PANTHERx Rare is a dual-accredited specialty pharmacy focused on rare and orphan disease, distributing several orphan products and providing access and support services to those needing them most. PANTHERx transforms lives by delivering medicine breakthroughs, clinical excellence, and access solutions to people living with rare and devastating conditions. While each rare condition affects few people, together all rare diseases impact an estimated 25 to 30 million Americans. Currently over 7,000 rare diseases have been identified and more than 90% of rare diseases are still without an FDA-approved treatment. Changes in federal policy and advances in science have led to a surge in FDA orphan drug approvals, providing tremendous hope to the rare disease community.

PANTHERx is a dual-accredited specialty pharmacy, holding distinctions in rare disease including the Accreditation Commission for Health Care (ACHC) Distinction in Orphan Drugs and Utilization Review Accreditation Commission (URAC) Rare Disease Center of Excellence. As a pharmacy focused on patient satisfaction, PANTHERx is now a six-time winner of the prestigious MMIT Patient Choice Award, including the 2023 honor. PANTHERx is headquartered in Pittsburgh, Pennsylvania and is licensed in all 50 states and US territories.

References: 

1. National Human Genome Research Institute. About Gaucher Disease | NHGRI. Genome.gov. Accessed January 26, 2024. https://www.genome.gov/Genetic-Disorders/Gaucher-Disease
2. National Gaucher Foundation. What Is Gaucher Disease? | National Gaucher Foundation. National Gaucher Foundation. Accessed January 26, 2024. https://www.gaucherdisease.org/about-gaucher-disease/what-is/
3. NORD. Gaucher Disease - NORD (National Organization for Rare Disorders). NORD (National Organization for Rare Disorders). Accessed January 26, 2024. https://rarediseases.org/rare-diseases/gaucher-disease/
4. Stirnemann J, Belmatoug N, Camou F, et al. A Review of Gaucher Disease Pathophysiology, Clinical Presentation and Treatments. Int J Mol Sci. 2017;18(2):441.
5. Bennett LL, Mohan D. Gaucher Disease and Its Treatment Options. ANN PHARMACOTHER. 2013;47(9):1182-1193.

For more information, please contact marketing@pantherxrare.com

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SOURCE PANTHERx Rare Pharmacy