- In May 2022, Ultragenyx Pharmaceuticals secured licensing rights for an experimental gene therapy aimed at treating Sanfilippo Syndrome. This acquisition strengthens their rare disease pipeline by incorporating innovative treatment options for the condition. The move highlights the company's focus on advancing gene therapy solutions for neurodegenerative disorders
- In January 2022, JCR Pharmaceuticals' drug JR-441 was granted "Orphan Drug" status in Europe for the treatment of Sanfilippo Syndrome. This designation provides the company with additional market exclusivity and regulatory incentives for developing the treatment. The status reinforces JCR's commitment to addressing rare diseases through innovative therapies
Frequently Asked Questions
The market is segmented based on Segmentation, By Diagnosis (GAG analysis, Activity Assay, Genomic DNA Sequencing, and Others), Disease Type (Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type III, Mucopolysaccharidosis Type IV A, Mucopolysaccharidosis Type VI, and Others), Treatment (Drugs, Enzyme Replacement Therapy, Gene Therapy, Genistein, and Others), Mode of Administration (Injectable, Oral, and Others), End User (Hospitals, Homecare, Specialty Clinics, and Others) – Industry Trends and Forecast to 2031
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The Global Sanfilippo A Market size was valued at USD 10.65 USD Million in 2023.
The Global Sanfilippo A Market is projected to grow at a CAGR of 9.3% during the forecast period of 2024 to 2031.
The market report covers data from the U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E., South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Brazil, Argentina and Rest of South America as part of South America.
