The first oral prescription for spinal muscular atrophy in China landed

Fudan Pediatrics participates in new drug research, "New England Journal of Medicine" releases latest research results

The first oral prescription for spinal muscular atrophy in China landed

Fudan Pediatrics participates in new drug research, "New England Journal of Medicine" publishes latest research results Fudan Pediatrics participates in new drug research, "New England Journal of Medicine" publishes latest research results Fudan Pediatrics participates in new drug research, "New England Journal of Medicine" publishes latest research resultsdomestic first The first oral prescription for spinal muscular atrophy in China landed

News from our newspaper (reporter Li Chenyan) Yesterday, the reporter learned from the Pediatric Hospital of Fudan University that the hospital had issued the first prescription for "Lisplan Oral Solution Powder" in China.
A 6-month-old type 1 spinal cord from Ningxia A female child with muscular dystrophy successfully received treatment, becoming the first child with spinal muscular atrophy receiving oral treatment in the country
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The global multi-center clinical trial FIREFISH of a new drug "Lisporan" for the treatment of rare spinal muscular atrophy jointly participated by Fudan Pediatrics has made new breakthroughs in the world, and the results were published in the New England Journal of Medicine recently
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Type 1 spinal muscular atrophy is a rare neurological disease that endangers multiple organs throughout the body and can cause disability and death.
It is caused by insufficient expression of motor neuron survivin.
Children often get onset within 6 months of age and cannot sit alone
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As the world's first small-molecule drug approved to directly target RNA, "Lisporan" can increase the level of functional motor neuron survival (SMN) protein through specific splicing of modified genes

FIREFISH is the first clinical research conducted in China for the treatment of spinal muscular atrophy
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According to Wang Yi, the leader of the Rare Diseases Group of the Pediatrics Branch of the Chinese Medical Association, one of the research project leaders, and a professor at the Children’s Hospital of Fudan University, the second part of the study will come from 41 people from 14 research centers in 10 countries1 -7-month-old children were used as the research object to evaluate the effectiveness and safety of "Lisporan" treatment

The results of the study showed that after 12 months of treatment, 29% of children achieved unsupported sitting alone for 5 seconds.
In the subsequent 24-month treatment data published, 61% of infants achieved sitting alone, and 78% of infants at 12 months.
Milestone breakthroughs in various degrees of sports have been achieved within
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Wang Yi said that in addition to the improvement in motor function, the 12-month survival rate of children has also been significantly improved, and the clinical data on the preservation of respiratory and swallowing functions are also very exciting

It is reported that in the FIREFISH study, Fudan Pediatrics contributed one-seventh of the cases.
The team members’ high-quality research execution, scale evaluation, and team collaboration capabilities ensure the smooth progress of the clinical trial and provide a reliable first for the clinical trial.
Hand research data, and make an important contribution to the successful research and development of new drugs
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This study also lays a solid foundation for future clinical trials of spinal muscular atrophy in Fudan Pediatrics, and further promotes the diagnosis, treatment, and research process of the disease