Rare Disease

News

US regulators reject Akcea and Ionis rare disease drug

The US Food and Drug Administration (FDA) rejected Akcea and Ionis’ application for the use of Waylivra in treatment of familial chylomicronemia syndrome (FCS) despite earlier support of ex

News

Amicus' oral Fabry disease drug approved in US

Decision follows approval in Europe

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He ain’t heavy – learning about a sibling’s DMD diagnosis

It’s common for patients to want to know more about what’s causing them to be ill and their DMD diagnosis and it’s the same for their families – however long that road may be.

Views & Analysis

LHON diagnosis: Opening peoples’ eyes to the invisible suffe...

Maryse Roger has firsthand experience of Leber’s hereditary optic neuropathy (LHON), having first assisted her two brothers as their sight deteriorated and then leading the care of her son

News

Genentech’s Rituxan approved for rare skin disease

The FDA has approved Genentech’s Rituxan (rituximab) for use in adults with moderate-to-severe pemphigus vulgaris (PV), making it the first treatment for the condition in more than 60 years.

News

NICE recommends Pfizer’s Xalkori in rare lung cancer

NICE has issued a Final Appraisal Determination that recommends the use of Pfizer’s Xalkori (crizotinib) in England under the Cancer Drugs Fund (CDF) for patients with ROS1-positive advanced non-small cell lung cancer (NSCLC).