Pancreatic exocrine insufficiency (PEI) is a condition where the pancreas does not produce enough digestive enzymes, leading to difficulties in digesting food and absorbing nutrients. This condition ...

Regenxbio's RGX-121 gene therapy for Hunter syndrome is the main catalyst, with FDA approval targeted for February 2026.

Austin, Aug. 04, 2025 (GLOBE NEWSWIRE) -- Enzyme Replacement Therapy Market Size & Trends: According to SNS Insider, The Enzyme Replacement Therapy Market was estimated at USD 11.27 billion in 2024 ...

A new study led by University of Pittsburgh and UPMC Hillman Cancer Center researchers shows that an enzyme called PARP1 is involved in repair of telomeres, the lengths of DNA that protect the tips of ...

Four patients with infantile-onset Pompe’s disease received a single intravenous injection of an adeno-associated virus serotype 9 vector carrying codon-optimized complementary DNA encoding human acid ...

A research study recently published in the American Journal of Gastroenterology explains the role of pancreatic enzyme therapy (PERT) in reducing the frequency of acute pancreatitis (AP) in children ...

Qure's early AMT-191 trial data in Fabry disease showed strong enzyme activity, manageable safety, and patient independence from enzyme replacement therapy.

Studies into a rare nervous system disorder that causes dementia and early death in children have highlighted a possible treatment for the condition. Regular infusions of a key enzyme that is ...

All patients in the first cohort achieved between 27- to 208-fold increases in α-Gal A activity relative to mean normal level ~~ All patients ...

It’s a big day for Sanofi as the pharmaceutical giant welcomes two European drug approvals, both for rare enzyme deficiencies. One regulatory nod ushers in the first treatment in Europe for a ...