As the world's first SMA disease correction treatment drug, Nostradamus was approved by FDA in December 2016, and was listed in Chinese Mainland within two and a half years. It was included in the national medical insurance in December 2021, opening a new era of SMA treatment. Now, after more than six years on the market in China, Norsen has been widely used in children and adult patients with SMA, and China has become the country with the highest number of people receiving this treatment worldwide.

In order to better monitor the treatment efficacy of adult SMA patients, the Chinese SMA Adult Disease Registry Research has emerged. The project is led by Peking Union Medical College Hospital and collaborates with 12 centers across the country to enroll 200 adult patients aged 18 and above with 5q SMA. It was launched in December 2022 and plans to follow up for 5 years. At present, this study has repeatedly presented the efficacy and safety data of Nordenafil in 5q SMA adult patients in China at domestic academic conferences and the 2025 MDA International Academic Conference.

At the MDA conference held in the United States from March 16-19, 2025, the latest analysis data based on type 2 and type 3 patient populations were presented in the Chinese SMA Adult and Child Disease Registry Study, further verifying the efficacy and safety of Norxanol in the treatment of 2/3 type 5q SMA adult and child patients, providing important evidence for clinical application.

The latest analysis released this time included 168 patients with type 2/3 SMA, including 37 patients with type 2 SMA and 131 patients with type 3 SMA. The median age of onset of treatment with Nordersen was 23 years (type 2 SMA) and 26 years (type 3 SMA), respectively. Research shows that at the 14th month, the overall response rates of SMA3 type walkable, type 3 type reclining, and type 2 reclining patients were 73.1%, 68.8%, and 60.0%, respectively. In addition, the HFMSE and RULM scores of most patients have improved compared to baseline, and patients with different motor function states have the potential for improvement. Even adult patients with severe symptoms have the opportunity to achieve significant functional improvement through treatment.