Business, energy, technology, markets and global industry news from Business News Today

lysosomal storage disorder

PharmaDeviceNews lysosomal storage disorder
Representative image: A pediatric neurology research setting highlights the clinical focus on tralesinidase alfa, enzyme replacement therapy, and long-term treatment advances for Sanfilippo syndrome type B.
Pharma & Biotech

Could Spruce Biosciences finally change the Sanfilippo syndrome type B treatment landscape?

Spruce Biosciences’ MPS IIIB data raise a pivotal question: can tralesinidase alfa turn biomarker gains into disease-changing therapy?

Read More
Representative image of a rare disease neurology consultation, illustrating how Sanofi’s venglustat FDA priority review could change treatment options for type 3 Gaucher disease.
Pharma & Biotech

Can Sanofi’s venglustat priority review change the treatment outlook for type 3 Gaucher disease?

Sanofi’s venglustat could target neurological type 3 Gaucher disease. The FDA review now tests whether rare disease care can shift.

Read More
Pharma & Biotech

IntraBio leverages prior Niemann-Pick approval to seek FDA expansion of levacetylleucine into Ataxia-Telangiectasia

IntraBio files the first-ever FDA application for an A-T therapy. Analyse the Phase III evidence, regulatory pathway, and what approval would mean.

Read More
Pharma & Biotech

Can AB-1009 reshape treatment for late-onset Pompe? AskBio prepares for first patient in U.S.

AskBio’s AB-1009 gene therapy for late-onset Pompe secures FDA IND clearance. Find out what this milestone means for AAV innovation and patient outlook.

Read More