Protected: Finding the roadmap for living with LHON
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Protected: Finding the roadmap for living with LHON
https://pharmaphorum.com/disease-spotlight/finding-the-roadmap-living-with-lhon/
rare disease
Amicus’ oral Fabry disease drug approved in US
Decision follows approval in Europe
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.
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).
FDA approves new treatment for rare genetic disease PKU
Novel enzyme therapy will treat adults with PKU who have uncontrolled blood phenylalanine concentrations with current treatment.
Roche haemophilia data piles pressure on Shire
Hemlibra threatens to take market share from Advate
