Geneneck announces the latest results of SMA therapy in clinical trials

recently, Roche-owned Genentech announced the latest results in two clinical trials of its oral spinal muscular dystrophy (SMA) therapy.test results show that risdiplam can not only improve the symptoms of patients with type 1 SMA, but also be used to treat patients with type 2 and type 3 SMA.about Risdiplam Risdiplam is an oral SMN2 RNA shear regulator developed jointly by Genente and PTC Therapeutics and the SMA Foundation.Risdiplam relieves patient symptoms by regulating the cutting process of SMN2 RNA to increase the level of mRNA that produces normal SMN proteins. The strategy for treating SMA is very similar to that of Spinraza, which has already been approved. The difference is that Spinraza uses anisotropy oligonucleotides (ASOs) to regulate RNA shearing, which needs to be injected directly into the cerebrospinal fluid. Risdiplam, on the other, is a small molecular drug that can be used or so on.study patients with type 1 SMA were treated with different doses of risdiplam in a clinical trial called FIREFISH. Part 1 of the trial is a dose increment study, while in part 2 of the trial, the patient is treated with the expected therapeutic dose of risdiplam. Part 2 results showed that after 12 months of treatment, 7 infants (41.2%) were able to sit independently for more than 5 seconds, 9 infants (52.9%) were able to maintain head integrity, and 1 infant (5.9%) reached the athletic ability milestone.in sunFISH clinical trials, patients with type 2 and type 3 SMA between the ages of 2 and 25 were treated with risdiplam. The trial is also divided into two parts, the first part is the dose increment study. After 12 months of treatment, the patient's SMN protein expression levels increased by more than 1 times on average. An assessment of the motor ability of patients who participated in the Part 1 study using the MFM32 scale showed that 58% of patients had an MFM32 score that was at least 3 points higher than the baseline. (Drug Information Network)