News

Mucopolysaccharidosis Type I Pipeline Appears Robust With 8+ Key Pharma Companies Actively Working in the Therapeutics Segment | DelveInsight
Promising Mucopolysaccharidosis Type I Pipeline Therapies such as Laronidase, Antihistamine, Antipyretic, JR-171, AGT-181, ...
Bone marrow transplant changes two-year-old bedridden Adhiraj’s life
Adhiraj Gaikwad, a two-year-old with two rare genetic disorders, received a bone marrow transplant in Mumbai to treat Mucopolysaccharidosis Type 1 (MP ...
The Kingston Whig-Standard6d
Kingston to be lit up in blue and yellow in support of rare genetic disorder
To help raise awareness about MPS, Kingston City Hall and Springer Market Square will be lit up in blue and yellow on May 15.
Pharmabiz6d
US FDA accepts and grants priority review status to Regenxbio’s BLA for clemidsogene lanparvovec to treat Mucopolysaccharidosis II
US FDA accepts and grants priority review status to Regenxbio’s BLA for clemidsogene lanparvovec to treat Mucopolysaccharidosis II: Rockville, Maryland Thursday, May 15, 2025, 1 ...
NS Pharma, Inc.: FDA Accepts Biologics License Application for Mucopolysaccharidosis II Treatment
NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku) announced today that the U.S.
FDA Accepts Biologics License Application for Mucopolysaccharidosis II Treatment
NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku) announced today that the U.S. Food and Drug Administration has accepted for review the Biologics License ...
The Post-Journal7d
Raising Awarenes: City Pastor, Family Raise Awareness Of MPS
Thursday, May 15, is MPS Awareness Day, a time to spread the word about this group of ultra rare diseases. When Jamestown ...
Regenxbio stock rises on FDA’s priority review for MPS II therapy
Investing.com -- Shares of Regenxbio Inc . (NASDAQ: RGNX) climbed 7.2% in premarket trading after the company announced the U.S. Food and Drug Administration (FDA) has accepted for priority review its ...
Immusoft to Present Data at ASGCT 2025, Demonstrating Positive Results from the World's First Engineered B Cell in a Human Clinical Trial
Immusoft of CA, a cell therapy company dedicated to improving the lives of patients with diseases modifiable with therapeutic proteins, today announced that Paul J. Orchard, MD., Professor, Division ...
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Mucopolysaccharidosis Type I Market to Witness Upsurge in Growth at a CAGR of 8% During the Forecast Period (2025-2034) | DelveInsight
Mucopolysaccharidosis Type I Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage ...