Lysosomal Storage Diseases

News

Ultragenyx gets FDA nod for first Sanfilippo A gene therapy

Patients in the US with Sanfilippo syndrome type A have their first FDA-approved therapy, Ultragenyx Pharma's $3.9m one-shot gene therapy Fayuvi.

News

November date set for FDA decision on Sanofi's Gaucher drug

The FDA has started a priority review of Sanofi's brain-acting Gaucher disease therapy venglustat, with an action date of 25th November.

News

FDA clears Denali's 'game-changer' Hunter syndrome drug

Denali's Avlayah is the first drug for neurological complications associated Hunter syndrome to be approved in the US.

News

Mixed data mars Sanofi's venglustat programme

Sanofi has reported positive phase 3 results with venglustat in Gaucher disease, but the drug missed the mark in a Fabry disease trial.

News

Azafaros bags €132m for rare neuro-metabolic disorder drugs

Azafaros is set to start phase 3 trials of its lead drug for a trio of rare lysosomal storage disorders, backed by €132m in new Series B financing.

Digital

Digital health technologies and rare diseases: Enhancing the...

This pre-recorded webinar was fully funded by Chiesi GRD and hosted by pharmaphorum. Professor Maurizio Scarpa received an honorarium for his participation and contribution to this webinar.

Ultragenyx gets FDA nod for first Sanfilippo A gene therapy

November date set for FDA decision on Sanofi's Gaucher drug

FDA clears Denali's 'game-changer' Hunter syndrome drug

Mixed data mars Sanofi's venglustat programme

Azafaros bags €132m for rare neuro-metabolic disorder drugs

Digital health technologies and rare diseases: Enhancing the...

Iambic prices its IPO, seeking to raise up to $160m

Discover a new era of healthcare panel engagement: Grounded ...

Lysosomal Storage Diseases

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