A 23-year-old woman with juvenile-onset α-mannosidosis developed an axonal polyneuropathy more than a year following successful unrelated donor (URD) BMT complicated by chronic graft-versus-host ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) approved ...

Researchers have developed an innovative, experimental gene transfer-based treatment for children with giant axonal neuropathy (GAN). This extremely rare genetic disorder causes children to gradually ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the U.S. Food and Drug Administration (FDA) has granted ...

A CRISPR-Cas9-based therapy targeting the gene encoding transthyretin greatly reduced transthyretin levels in patients with ...

The latest reports come from 2 separate European groups. Dr C Metzler (University Hospital of Schleswig-Holstein, Luebeck, Germany) and colleagues told the British Society of Rheumatology meeting how ...

What is the recommended approach to managing a patient infected with hepatitis C virus who has been on combination interferon and ribavirin therapy and who has developed peripheral neuropathy?

"Alterations in axonal membrane and channel function occur early in the disease course, prior to discernable large fibre involvement, and has the potential to be used as an early biomarker of ...

Abnormal glucose metabolism, which occurs when the body has difficulty processing sugar (glucose) into energy, is twice as common among patients with chronic nerve dysfunction of unknown cause than ...

Half of patients referred to a specialist centre with a diagnosis of chronic inflammatory demyelinating polyneuropathy (CIDP) do not actually have the condition, report researchers. Of 58 patients ...

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare, acquired neurological disorder with heterogeneous presentation. It is a peripheral nerve system immune-mediated condition which ...