Genentek's oral SMA therapy has resulted in positive clinical results

today, Roche-owned Genentech today announced the latest results in two clinical trials of its oral spinal muscular dystrophy (SMA) therapy. The 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. Following the release yesterday of the latest clinical data from Zolgensma, a gene therapy for SMA, Novarma's results suggest that future SMA patients may have a variety of innovative treatment options.SMA is a serious neuromuscular disease in which patients suffer from aggressive muscle weakness and paralysis due to the death of motor neurons. SMA is due to mutations in the SMN1 gene that encodes motor neurosurgery protein (SMN), resulting in the loss of SMN protein levels. SMA patients may carry different copies of the SMN2 gene, which causes RNA clipping errors due to mutations that produce only 10% of the mRNA that produces normal SMN proteins. Usually the more copies of the SMN2 gene smas that SMA patients carry, the less symptoms SMA symptoms are.Risdiplam is an oral SMN2 RNA shear regulator developed jointly by Genentek and PTC Therapeutics and the SMA Foundation. By regulating the cutting process of SMN2 RNA, it increases the level of mRNA that produces normal SMN proteins, thereby alleviating the patient's symptoms. 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.in a clinical trial called FIREFISH, patients with type 1 SMA were treated with different doses of risdiplam. 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.the company plans to include the latest clinical trial data released today in regulatory filings with the FDA and the European Medicines Agency (EMA). Applications are expected to be submitted in the second half of 2019.we wish this new drug a smooth development and innovative treatment options for SMA patients. (Drug Ming Kant)