Rare Disease

News

CHMP backs Sanofi's Xenpozyme as first drug for ASMD

Sanofi's Genzyme unit has moved a step closer to securing EU approval for Xenpozyme – its enzyme replacement therapy for rare disease acid sphingomyelinase deficiency (ASMD) – after scoring

R&D

Big pharma vs small diseases: Tackling rare diseases

Rare diseases aren’t as rare as the appellation suggests.

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Webinars

Medical Informatics and Rare Disease: a bridge between two w...

With more than 6,000 different diseases comprising the rare disease (RD) landscape, connecting patients and practitioners with the expertise needed to achieve th

R&D

Does the answer to advancing rare disease treatment lie in r...

What do 300 million people have in common? They are living with rare diseases that are defined by their scarcity within the general population.

R&D

Medical Informatics in Rare Disease – the bridge between two...

Rare diseases bring with them a multitude of challenges for those affected, their relatives and care providers.

News

FDA clears Marinus' Ztalmy as first drug for rare epilepsy

People with CDKL5 deficiency disorder (CDD) now have their first FDA-approved treatment, after the US regulator gave the nod to Marinus Pharma's Ztalmy for the rare genetic form of