Heightened clinical activity and pipeline advancement highlighted by Quince Therapeutics’ Phase 3 NEAT trial evaluation of eDSP, IntraBio’s Fast Track development of IB1001, Acasti Pharma’s ...

Quince Therapeutics has disclosed outcomes from the Phase III ATTeST clinical trial of EryDex (dexamethasone sodium phosphate encapsulated in autologous erythrocytes) for treating ...

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 ...

A Prescription Drug User Fee Act target date of September 19, 2026 has been assigned to the application. The Food and Drug Administration (FDA) has granted Priority Review to the supplemental New Drug ...

Levacetylleucine Could Become the First-Ever FDA-Approved Treatment for Ataxia-Telangiectasia FDA accepts sNDA for AQNEURSA® (levacetylleucine) for Ataxia-Telangiectasia — a rare, progressive ...

Ataxia-telangiectasia (A-T) is an autosomal recessive disorder characterized by cerebellar ataxia, telangiectasias, immune defects and a predisposition to malignancy. The birth frequency of A-T is ...

Revised Risk Classification for Pediatric Extracranial Germ Cell Tumors Based on 25 Years of Clinical Trial Data From the United Kingdom and United States B-cell NHL, HL, and acute lymphoblastic ...

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 ...

Ataxia telangiectasia (AT) is a genetic multisystem disorder, presenting with progressive ataxia, immune deficiency, and propensity toward malignancy. Endocrine abnormalities (growth retardation, ...

Rutgers scientists think they have found a way to prevent and possibly reverse the most debilitating symptoms of a rare, progressive childhood degenerative disease that leaves children with slurred ...

IntraBio Inc. today announced that results from its pivotal Phase III clinical trial evaluating levacetylleucine for the treatment of Ataxia-Telangiectasia (A-T) have been published in The Lancet ...

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