China News Service, Xiamen, March 25th (Yang Fushan and Shi Qingqing) "Doctor, do I have strength in my hands and feet after this injection?" A Hua (pseudonym), a patient with the rare disease "Spinal Muscular Atrophy" 24 On the 25th, he was successfully injected with the life-saving drug Nosinason Sodium in Zhongshan Hospital Affiliated to Xiamen University, and was discharged from the hospital on the 25th.
Zhongshan Hospital Affiliated to Xiamen University said that the drug is the world's first precision targeted therapy drug for the treatment of spinal muscular atrophy. It has entered the new version of the National Medical Insurance Drug List through national medical insurance negotiation, and the price has increased from the original nearly 700,000 yuan per injection. , down to more than 30,000 yuan per needle.
Ahua is the first adult patient in southern Fujian to benefit.
Spinal muscular atrophy (SMA) is a neurodegenerative disease that causes progressive, symmetrical muscle weakness and muscle atrophy in proximal limbs and trunk due to degeneration of the anterior horn of the spinal cord and medulla oblongata motor neurons. Infants with the more severe form of the disease (SMA type I) do not even survive to age two without respiratory intervention.
Other types of patients may eventually lose the ability to walk due to progressive disease progression, and develop breathing and swallowing disorders, which bring a heavy burden to the family and society.
The hospital said that treating this rare disease is a world-class problem.
A 27-year-old patient, Ahua, developed bilateral lower extremity weakness 14 years ago. Later, he was diagnosed with SMA and sought medical treatment in many places.
During the long 14 years, Ahua gradually developed muscle atrophy in his limbs, unable to run, and even had to rely on handrails to go up and down stairs.
It was not until the emergence of Nosinaxen that was included in medical insurance that ordinary people like Ahua had the possibility of recovery.
The diagnosis and management of spinal muscular atrophy requires multidisciplinary collaboration.
In order to ensure that Ahua's treatment is foolproof, multidisciplinary experts from Zhongshan Hospital Affiliated to Xiamen University made a comprehensive analysis and all-round evaluation, formulated a comprehensive treatment and administration plan, selected the most economical medical expenses, and introduced the first domestic injection of SMA specific drugs. The team of Professor Chen Wanjin from the First Affiliated Hospital of Fujian Medical University "learned lessons" and made every effort to escort the health of patients.
Chen Xingyu, director of the Department of Neurology, Zhongshan Hospital Affiliated to Xiamen University, said that the successful development of this case has ushered in the era of gene therapy for neurogenetic diseases in southern Fujian.
The hospital said that next, the department will continue to carry out scientific research with the team of Professor Chen Wanjin of the First Affiliated Hospital of Fujian Medical University, and establish the first SMA multi-center cerebrospinal fluid bank in the country, in order to benefit more patients.
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