China to carry out adult freezing disease injection treatment

Recently, Beijing Concord Hospital for 2 cases of spinal muscular dystrophy adult patients under the ultrasound-guided gene therapy of the new drug intra-drug injection treatment, the patient's condition is stable. This is the first time in China for adult patients to carry out intra-intravenous injection therapy, but also the first time the ultrasound visualization technology used in intra-intra-intravenous injection treatment Adult drug intra-drug injections are more difficult and at higher risk of uncertainty than infants and young children. The successful implementation of this treatment will accumulate more experience for the treatment of spinal muscular dystrophy in adults.Spinal muscular dystrophy is a rare inherited neuromuscular disease, which is a "frozen disease" of childhood disease. Nosinsheng injection is the world's first approved gene therapy drug for spinal myotrophy, early this year through China's rare disease drug priority review and approval process in China, in October this year officially used in domestic patient treatment.The first to try the new drug at Beijing Concord Hospital was a 34-year-old female patient and a 28-year-old male patient who had been in a wheelchair for more than 20 years when they were 1 and a half years old and 3 months old, and who were currently only slightly active in the upper limbs and had severe scoliosis.
The hospital organized a multidisciplinary team to conduct a comprehensive examination of the patient to assess possible punctures into the road. Recently, the hospital's neurology, radiology, anesthesiology, physical medicine rehabilitation, digestive medicine, respiratory medicine, basic surgery, orthopaedics, cardiology, ultrasound medicine, pharmacy and other related departments and out-of-hospital experts focused on the two patients' condition and medication. In the afternoon, the treatment team successfully performed ultrasound-guided lumbar puncture and intra-lumbar drug injections for two patients in the outpatient operating room.
During the course of treatment, the patient was completely unable to maintain a stable position and was treated with the assistance of medical staff. Anesthesiologists through ultrasound repeatedly carefully and patiently groping, in the severely deformed spinal gap to find the best puncture into the road, completed a very difficult dosing operation. Dai Yi, a neurologist at the hospital, said: "The lumbar puncture of the normal spine is a routine operation. However, these two patients due to the long-term progression of the disease caused severe scoliosis of the spine, can not carry out regular waist wear. Ultrasound guide under the waist wear is also very difficult, and finally from the unconventional position and special angle before the puncture success. Although there are adult patients abroad have done intra-intra-thyotherapy, but ultrasound-guided intra-drug treatment has not been reported.
(Health Journal)