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A long-established drug with a diverse medical history is drawing new attention for its potential role in treating an ...
Genethon, a leading laboratory in gene therapy for rare diseases, today announced that it has entered into an exclusive, worldwide licensing agreement with AskBio, a gene therapy company wholly owned ...
In this open-label phase 1–2 study, pediatric patients with mucopolysaccharidosis type II treated with tividenofusp alfa showed substantial reductions in cerebrospinal fluid and urinary heparan ...
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Mom reveals the subtle sign her kids had 'childhood dementia'
A mom reveals the one subtle sign that led to both her children being diagnosed with a condition dubbed ‘childhood dementia’.
A Muskego family helped raise more than $5.5 million to fund early access treatment for children with Sanfilippo syndrome ...
The FDA previously declined to approve UX111 citing CMC-related observations, but the firm is hopeful the agency will approve its new regulatory application.
Ultrafine plastic microparticles have been detected in ocular compartments, raising concern about their role in degenerative eye diseases. Nevertheless, significant efforts are required to elucidate ...
At the mRNA Health conference in Berlin, enginzyme and AGC Inc. presented a scalable process to produce a key mRNA vaccine and therapy ingredient, N1-methylpseudouridine-5'-triphosphate (m (1) TP).
After greenlighting 56 novel therapeutics in 2025, four notable applications continue to await the agency’s action after ...
A large team of researchers led by Wouter Karthaus, head of the Endocrine Therapy Resistance and Molecular Genetics Lab at ...
Intrapleural enzyme therapy (IET) improves outcomes in adult inpatients with complicated parapneumonic pleural effusion or empyema.
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