Phase 3 trial results show levacetylleucine improves SARA scores in patients with the rare disorder ataxia-telangiectasia.

If approved, levacetylleucine could become the first therapy on the market for ataxia-telangiectasia.

Backed by more than a dozen prominent investors, the startup is advancing into human testing a drug it believes capable of treating two disparate conditions, one of which is the target of Merck’s fast ...

Affecting the nervous system, immune system and other body organs, ataxia telangiectasia is characterized with uncoordinated movements of the individual suffering from this disorder. The secondary ...

Hereditary hemorrhagic telangiectasia, or Osler–Weber–Rendu syndrome, is an autosomal dominant vascular disorder that affects multiple systems. It is characterized by skin and mucosal telangiectasias ...

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Please provide your email address to receive an email when new articles are posted on . The NT-501 implant demonstrated positive results in two replicative phase 3 clinical trials for the treatment of ...

A total of 76 participants have been enrolled, including 65 participants in the six to nine year-old primary analysis population and 11 participants aged 10 years or older. Study powering based on ...

An IgE deficiency was demonstrated in 11 of 16 patients with ataxia-telangiectasia. No relation to age or to the level of IgG, IgM or IgD was found. Nine out of 11 patients with IgE and IgA deficiency ...

The Food and Drug Administration (FDA) has extended the review period for the Biologics License Application (BLA) for NT-501 (revakinagene taroretcel) in the treatment of macular telangiectasia type 2 ...

IMMUNOLOGIC abnormalities, including hypogammaglobulinemia, abnormalities of gamma 1 A globulin, poor homograft rejection and structural abnormalities of the thymus gland, have recently been reported ...