Rare Disease

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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.

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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

News

NICE firms up NHS use of Vimizim for Morquio A syndrome

In 2015, NICE said that BioMarin's Vimizim could be used to treat patients with the ultra-rare disease Morquio A syndrome, while real-world data was collected on its efficacy.

News

Illumina debuts its multi-gene test for cancer in Europe

Gene sequencing specialist Illumina has started rolling out a new test that looks for a battery of tumour-associated genes from a single tissue sample, and could be used to help guide treat

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

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

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

FDA clears Marinus' Ztalmy as first drug for rare epilepsy

NICE firms up NHS use of Vimizim for Morquio A syndrome

Illumina debuts its multi-gene test for cancer in Europe

CDER head Tidmarsh 'on leave' and is sued by drugmaker

How to meet your physicians where they are online

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