RSVP Live on MSN

Donegal boy (10) is one of six people in the world to be diagnosed with rare disease

Jason Brady ended up in the hospital around fifty times by the time he turned two years old, and he was officially diagnosed ...
Belfast Live on MSN

Ten-year-old Donegal boy one of six people in the world diagnosed with rare disease

He ended up in the hospital around 50 times by the time he turned two and was officially diagnosed at age seven ...
pharmaphorum

CHMP backs Biogen’s Skyclarys for Friedreich’s ataxia

Biogen’s Skyclarys is on course to becoming the first approved medicine for the inherited neurological disease Friedreich’s ataxia (FA) in the EU after it was recommended by the EMA’s human medicines ...
pharmaphorum

Biogen bids to extend use of Skyclarys to younger patients

Biogen has started a phase 3 trial that it hopes will extend the label for its Skyclarys therapy for inherited neurological disease Friedreich's ataxia (FA) to include children as young as two.
Nature

The Huntington's disease-like syndromes: what to consider in patients with a negative Huntington's disease gene test

Huntington's disease (HD), which is caused by a triplet-repeat expansion in the IT15 gene (also known as huntingtin or HD), accounts for about 90% of cases of chorea of genetic etiology. In recent ...
Nature

Monoclonal antibody and intravenous immunoglobulin therapy for rheumatic diseases: rationale and mechanisms of action

Advances in our understanding of the pathogenesis of rheumatic diseases such as rheumatoid arthritis and systemic lupus erythematosus have led to the emergence of immunoglobulin-based therapy as a ...