Nya studieresultat bekräftar effekten med Spinraza vid SMA typ 2 eller 3

Nya studieresultat bekräftar effekten med Spinraza vid SMA typ 2 eller 3. NURTURE studier utförda med SPINRAZA-behandlade ungdomar/ unga vuxna med SMA typ 2 och 3 (17-19 år) visade stabil eller förbättrad motorfunktion och förbättrad livskvalitet
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Biogen (Nasdaq: BIIB) today announced new interim Phase 2 results from NURTURE, the ongoing open-label, single-arm study evaluating the efficacy and safety of SPINRAZA® (nusinersen) among pre-symptomatic infants with spinal muscular atrophy (SMA). In NURTURE, all infants treated with SPINRAZA were alive, did not require permanent ventilation and showed improvement in motor function and motor milestone achievements as of July 5, 2017, compared to the disease’s natural history. This study, along with a case series demonstrating SPINRAZA’s effectiveness among teens and young adults, will be presented at the Muscular Dystrophy Association (MDA) Clinical Conference on March 11-14, 2018, in Arlington, Virginia.

“These results reinforce SPINRAZA’s effectiveness as the first and only approved treatment for SMA and demonstrate once again the benefit it can bring to individuals with SMA, including infants, teens and young adults,” said Alfred Sandrock, M.D., Ph.D., executive vice president and chief medical officer at Biogen. “SPINRAZA is supported by the largest well-controlled SMA clinical development program conducted to date and we are committed to ongoing research in SMA. Biogen looks forward to working with healthcare providers and institutions in order to provide teens and young adults with SMA access to SPINRAZA, a treatment that has demonstrated significant benefit in the maintenance of motor function for these patients.”

Nya kliniska studier: Spinraza förbättrar motor och respirator funktion vid SMA-barn,

An early treatment with Spinraza improves motor function of patients with spinal muscular atrophy

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One Response to Nya studieresultat bekräftar effekten med Spinraza vid SMA typ 2 eller 3

  1. Ambulatory function and fatigue in nusinersen-treated children with spinal muscular atrophy typ 2 and typ 3

    Improvements in mean Hammersmith Infant Neurological Examination (HINE) motor milestones scores vs Baseline have been observed. Infants have generally gained weight over time consistent with normal development, and mean CMAP scores have mostly improved vs Baseline. There were no severe AEs reported, 5 infants experienced a serious AE. Three infants experienced AEs considered possibly related to study drug. No new safety concerns were identified.