Dansk neurolog planlægger at primær SMA-screenin til nyfødte

Mens andre lande allerede har dette, men Dansk neurolog vil indføre screening for nyfødte, så de hurtigt kan få behandling. Desværre er Spinraza i Danmark kun tilgængelig for børn op til 6 år

Han har sammen med en gruppe kolleger for nylig sendt et forslag til Sundhedsstyrelsen om at arbejde for at indføre neonatal screening for SMA i Danmark. Forslaget står foreløbig til at blive taget op på et møde i et udvalg i Sundhedsstyrelsen i løbet af foråret, og derefter vil det vise sig, om lægerne får opbakning til ønsket.

Risdiplam demonstrated statistically significant and medically meaningful motor milestone improvement in infants with Type 1 SMA

Basel, 23 January 2020 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced positive topline results from the pivotal Part 2 of the FIREFISH study, evaluating risdiplam in infants aged 1-7 months with Type 1 spinal muscular atrophy (SMA).

The primary outcome measure of the study was the proportion of infants sitting without support for at least five seconds at 12-months of treatment, assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). Safety for risdiplam in the FIREFISH study was consistent with its known safety profile and no new safety signals were identified. To date, more than 400 patients have been treated with risdiplam across all studies, with no treatment related safety findings leading to study withdrawal in any risdiplam trial

Material provided Roche

NCT02913482 (Firefish)

Roche announced that FDA grants priority review to risdiplam for spinal muscular atrophy

Wie start the week with a great news!

Roche announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) and granted Priority Review for risdiplam, an investigational survival motor neuron-2 (SMN-2) splicing modifier for SMA. Risdiplam is designed to increase and sustain SMN protein levels both throughout the central nervous system and peripheral tissues of the body. The FDA is expected to make a decision on approval by May 24, 2020.

“The FIREFISH and SUNFISH trials were designed to represent the real world spectrum of people living with SMA and include many people previously underrepresented in clinical trials,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “We look forward to working closely with the FDA to explore broad access to risdiplam for all individuals in the community who might benefit.”