In children with MS, ocrelizumab's safety and pharmacokinetic profile are similar to those in adults.

Published Date: 02 Jun 2024

There were no clinical relapses in pediatric patients receiving ocrelizumab, and their safety profile was comparable to earlier reports.

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Findings from the phase 3 TransportNPC study showed that Trappsol Cyclo missed the primary end point in Niemann-Pick type C, but subgroup and survival data support planned 2026 NDA.

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The Accelerating Access to Critical Therapies for ALS Reauthorization Act of 2026 extends federal support for ALS research and expanded access to investigational therapies, with additional provisions addressing clinical trial oversight and regulatory planning.

The professor of neurology at the University of Lyon and 2026 Rachel Horne Prize Winner discussed pregnancy counseling, therapeutic inertia, and overlooked women's health issues in multiple sclerosis.

Real-world 12‑month results from WeSMA, a phase 4 study, showed that oral risdiplam is well tolerated in adults with SMA, with stable motor function and consistent safety findings.

A phase 4 study is evaluating whether adults with chronic inflammatory demyelinating polyneuropathy can transition from intravenous immunoglobulin to subcutaneous efgartigimod PH20 without first experiencing disease worsening.

A new phase 4 trial, OCTAGON, will test a rapid, predefined corticosteroid-tapering schedule in patients with generalized myasthenia gravis treated with ravulizumab.

New 12-month interim data showed functional gains with BBP-418 vs placebo, consistent benefit across genotype, age, and baseline pulmonary function subgroups, and a safety profile comparable to placebo.

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The positive opinion for intravenous ocrelizumab was supported by phase 3 OPERETTA 2 findings demonstrating noninferior relapse control and superior suppression of MRI-detected brain lesions compared with fingolimod in pediatric patients with relapsing MS.

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