ultra-rare diseases

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Ultragenyx wins FDA approval for first treatment targeting r...

Ultragenyx secured its first gene therapy approval, the FDA clearing GENGLYCOS as the first treatment designed to address underlying cause of GSDla.

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UK patients get early access to Alnylam's ultra-rare disease...

The UK's regulator has granted early access to Alnylam's ultra-rare disease drug lumasiran, allowing patients with primary hyperoxaluria type 1 to gain access ahead of a regulatory decision

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Germany first in EU to get Novartis' SMA gene therapy, costi...

Novartis is set to launch its one-off gene therapy Zolgensma for the ultra-rare muscle-wasting disease spinal muscular atrophy (SMA) in Germany at the start of July priced at 1,945,000 euro

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Ionis' FCS drug approved in EU, launch due in Germany

Ionis Pharmaceuticals’ RNA-targeted drug Waylivra has been conditionally approved in Europe for the devastating ultra-rare disease Familial Chylomicronemia Syndrome (FCS).

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NICE rejects Alnylam and Akcea's amyloidosis drugs

NICE has rejected two pioneering drugs for the rare inherited condition, hereditary transthyretin-related amyloidosis (hATTR) in first draft guidance.

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