A novel indwelling subcutaneous intrathecal catheter circumvented the complex spine anatomy of patients with spinal muscular atrophy (SMA), a challenge when giving nusinersen via intrathecal injection ...

Mucopolysaccharidosis VI (MPS-VI) is caused by a deficiency in N-acetylgalactosamine-4-sulfatase activity, resulting in lysosomal accumulation of partially degraded glycosaminoglycans (GAGs).

The approval was supported by data from phase 3 STEER study, which enrolled patients with SMA who were treatment-naive and able to sit but never able to walk independently.

ALTERNATIVE (EGF114299): A study of lapatinib, trastuzumab, and endocrine therapy in patients who received neo-/adjuvant trastuzumab (IV) and endocrine therapy. This is an ASCO Meeting Abstract from ...

Credit: FDA. The FDA has received reports of tranexamic acid injection being erroneously administered intrathecally in the perioperative setting instead of the intended intrathecal anesthetic (eg, ...

The findings, say the researchers, confirm that repeated intrathecal injections of the gene-targeting therapy are safe, adding to the limited data on the long-term effect and safety of the treatment ...

Differences in the distribution of methotrexate into high grade gliomas following intravenous administration, as monitored by microdialysis, are associated with blood brain barrier integrity No ...

Biogen has partnered with Alcyone Therapeutics to improve the delivery of its spinal muscular atrophy drug Spinraza, handing over $10 million upfront for a global license to an implantable device.

Researchers found that treating mice with a single spinal injection of a protein called AIBP -- and thus switching 'off' TLR4, a pro-inflammatory molecule -- prevented and reversed inflammation and ...

A preliminary investigation by the Chongqing Municipal Health Commission has found that a doctor at the Chongqing Medical ...

The Chongqing Municipal Health Commission said on Saturday that, following online reports alleging that Children’ s Hospital ...