Olivia had been showing symptoms since she was just 7 months old — including a belly that remained swollen, hard to the touch ...

Denali's lead drug for Hunter syndrome wins FDA Priority Review, marking a key step toward commercial-stage status.

Tividenofusp alfa is a novel central nervous system-penetrant enzyme replacement therapy designed to address the neurological manifestations of MPS II by delivering IDS into the brain and body.

Nasdaq compliance follows positive upward trend in Company's Stock Price Jupiter, Florida, July 10, 2025 (GLOBE NEWSW ...

Nasdaq compliance follows positive upward trend in Company's Stock Price Jupiter, Florida, July 10, 2025 (GLOBE NEWSWIRE) -- Jupiter Neurosciences, Inc. (NASDAQ: JUNS) ("Jupiter" or the "Company"), ...

--Jupiter Neurosciences, Inc., a clinical-stage pharmaceutical company developing JOTROL™, a patented resveratrol-based platform, is pleased to unveil Nugevia™ PWR, a daily mitochondrial ...

For example, the prevalence of the mucopolysaccharidoses group of metabolic disorders is 14 times higher in Saudi Arabia than in the United States, providing a significant market opportunity.

Martin Mackay, head of R&D at Alexion, talks to pharmaphorum about the company's research pipeline for severe and ultra-rare diseases. Alexion has become hugely successful in recent years thanks ...

Abstract Mucopolysaccharidoses (MPS) are rare inborn errors of metabolism, leading to the accumulation of glycosaminoglycans (GAG) in distinct tissues. We investigated oropharyngeal dysphagia using ...

Lysosomal storage diseases are a group of metabolic disorders caused by deficiencies of several components of lysosomal function. Most commonly affected are lysosomal hydrolases, which are involved in ...

### 2.1 - Primary Congenital Forms/Childhood Glaucomas Primary congenital glaucoma is a rare disease but has a major impact on the child’s development and quality of life over his/her whole life span.

This important work advances our understanding of CHMP5's role in regulating osteogenesis through its impact on cellular senescence. The evidence supporting the conclusion is convincing and the ...