Children with spinal muscular atrophy (SMA) type 1 who started treatment with Evrysdi (risdiplam) in the first months of life are continuing to gain motor skills after three years of treatment, according to new data from the FIREFISH trial. “These long-term results in babies treated with Evrysdi are very…

Chugai Pharmaceutical, part of the Roche Group, is asking that risdiplam be approved as a daily oral treatment for spinal muscular atrophy (SMA) patients in Japan. A new drug application seeking approval, submitted to that country’s Ministry of Health, Labour and Welfare (MHLW), may be given priority review…

Infants with spinal muscular atrophy (SMA) type 1 receiving the therapeutic dose of Evrysdi (risdiplam) continue to improve and achieve motor milestones, according to two-year data from the first part of the Phase 2/3 FIREFISH clinical trial. Updated data from FIREFISH (NCT02913482) showed that nearly all…

Genentech announced, without providing details, positive topline results from the pivotal second part of its FIREFISH clinical trial of risdiplam, now before the U.S. Food and Drug Administration for approval. The company said in its release that risdiplam demonstrated statistically significant and medically meaningful motor milestone improvement…

Risdiplam (RG7916) can improve both survival and motor function in patients with type 1 spinal muscular atrophy (SMA) beyond that expected in the natural history of the disease, according to recent clinical data. These data were presented during the 2019 annual meeting of the American Academy of…

The European Medicines Agency has granted a priority medicines, or PRIME, designation to oral therapy candidate risdiplam to treat spinal muscular atrophy (SMA). PRIME designation is intended to encourage the development of promising therapies by speeding their evaluation so that the therapies, if proven effective, can be available to patients…

Roche has stopped the development of olesoxime, a treatment candidate for spinal muscular atrophy (SMA) following disappointing 18-month results in an ongoing clinical study, the company announced. In a statement shared online through TreatSMA, Roche mentioned the most recent results of the OLEOS Phase 2 trial (…

A potential oral treatment for children with type 1 spinal muscular atrophy (SMA) called  RG7916 was able to safely raise levels of the crucial SMN protein that is lacking in these patients, early results of a two-part clinical trial show. Data from the study, “RG7916 significantly increases SMN Protein in…

Roche’s treatment candidate RG7916 increased the amount of full-length, functional SMN protein in spinal muscular atrophy (SMA) patients, according to soon-to-be-presented data from a Phase 2/3 clinical trial. The company will share its clinical results at the 2018 American Academy of Neurology (AAN) Annual Meeting, April 21-27 in Los…

Early interim data from a Phase 2 clinical trial evaluating varying doses of a potential oral therapy, RG7916, in infants with type 1 spinal muscular atrophy (SMA) is showing good safety and tolerability, researchers said in a recent scientific presentation. Preliminary findings of 13 babies enrolled…