Amyloidosis represents a heterogeneous group of disorders defined by the extracellular deposition of misfolded protein fibrils, which can cause severe impairment in multiple organs. In the context ...

CAMBRIDGE, Mass., June 09, 2025--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNA interference (RNAi) therapeutics company, today announced that the European Commission (EC) has ...

Systemic amyloidoses are potentially lethal diseases. A prerequisite for adequate treatment is a proper diagnostic and prognostic work-up.

Indications In the EU, AMVUTTRA ® (vutrisiran) is indicated for the treatment of: hereditary transthyretin amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy (hATTR-PN).

Florbetaben (18F) has been granted Orphan drug designation for the diagnosis of ATTR amyloidosisFlorbetaben (18F) previously received orphan drug designation as a diagnostic agent for the management ...

Previously known as primary amyloidosis, systemic amyloid light chain (AL) amyloidosis is the most common form of systemic amyloidosis in developed countries. Although occasionally localized and ...

Alnylam has been a pioneer in hereditary ATTR amyloidosis, getting approval in 2018 for the first drug to treat the rare disease, and has now launched a digital companion to help patients track ...

The FDA has accepted two separate marketing applications for the drug – both for cardiomyopathy associated with the rare disease ATTR amyloidosis – and awarded a priority review for one of them.

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