Intellia earlier this year reported a similar grade 4 liver enzyme elevation associated with the gene therapy nexiguran ...

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 ...

After Intellia Therapeutics paused a pair of phase 3 trials for its CRISPR therapy in response to a liver safety signal, the ...

Dublin, Oct. 02, 2025 (GLOBE NEWSWIRE) -- The "Mucopolysaccharidosis Market - A Global and Regional Analysis: Focus on Treatment, Disease Type, Route of Administration, End User, Country, and Region - ...

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 ...

Researchers from Drexel University’s College of Arts and Sciences and College of Medicine have found a potential new therapeutic target for Alzheimer’s disease. Expanding on their previous research on ...

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 ...

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 ...

The U.S. Food and Drug Administration has placed a clinical hold on Intellia Therapeutics' two late-stage trials testing an ...

BioMarin Pharmaceutical BMRN reported third-quarter 2025 adjusted earnings per share (EPS) of 12 cents against the Zacks ...

Preclinical study showed PDIA1 and PDIA5 protect cells from oxidative stress, maintain mitochondrial function, and are ...