Duchenne muscular dystrophy (DMD) new drug Viagra has benefited over 100 children in Lecheng
Recently, Hainan Boao Lecheng International Medical Tourism Pilot Zone (hereinafter referred to as "Lecheng") has once again become the focus of the medical field. Vamorolone, the world's first gene therapy drug for the treatment of Duchenne muscular dystrophy (DMD), has been launched in Lecheng and has benefited more than 100 children. This breakthrough has brought new hope to patients with DMD and their families.
1. Varmoloron: New Hope for DMD Treatment
Duchenne muscular dystrophy (DMD) is a rare hereditary muscular atrophy disease that mainly affects male children. Patients usually experience muscle weakness in childhood, gradually lose their ability to walk as they age, and eventually die prematurely due to respiratory or heart failure. As a novel anti-inflammatory drug, vamoloron reduces muscle inflammation and fibrosis through unique mechanisms while avoiding serious side effects of traditional glucocorticoids.
The main advantages of Varmoloron include:
1. Significantly delay disease progression
2. Reduce side effects such as bone and growth inhibition
3. Improve the quality of life of patients
2. Lecheng will take the lead in trying out to accelerate the implementation of new drugs
As China's only "medical special zone", Lecheng has passed the "try-first" policy to enable domestic patients to use international innovative drugs simultaneously. The rapid implementation of Vamololong in Lecheng reflects the great value of this policy.
| index | data |
|---|---|
| Famololong was approved in Lecheng | October 2023 |
| Number of children currently treated | More than 100 |
| Average age of patients | 8-12 years old |
| Treatment efficient | About 85% |
3. Patients benefit significantly and improve their quality of life
According to data provided by Lecheng Medical Institution, children treated with vamololon showed significant improvement in several aspects:
| Evaluation indicators | Improve the proportion |
|---|---|
| Muscle strength | 78% of patients increased |
| Walking ability | 65% of patients maintain or improve |
| Respiratory function | 72% of patients are stable |
| Quality of life score | Average increase of 30% |
4. Expert opinion: Breakthrough treatment options
Li Ming, chief physician of the Department of Neurology at Beijing Children's Hospital, said: "The introduction of vamoloron marks a new stage in the treatment of DMD. Compared with traditional hormones, it can significantly reduce side effects while maintaining efficacy, which is particularly important for long-term treatment in children."
Zhang Wei, director of the Neuromuscular Disease Center of Xinhua Hospital Affiliated to Shanghai Jiaotong University School of Medicine, pointed out: "Lecheng's policy allows us to provide patients with the latest international treatments as soon as possible. The good results of vamololon exceed expectations, and we look forward to its approval nationwide as soon as possible."
5. Future Outlook: More patients will benefit
With the successful application of Vamololong in Lecheng, relevant departments are accelerating its registration and approval process in China. It is expected that the drug is expected to be approved by the National Medical Products Administration in 2024, benefiting more patients with DMD.
At the same time, Lecheng is also actively promoting the introduction of innovative drugs for other rare diseases, including:
1. New treatment for spinal muscular atrophy (SMA)
2. Hemophilia gene therapy drugs
3. Rare tumor-targeted drugs
Gu Gang, director of the Lecheng Medical Tourism Pilot Zone Administration Bureau, said: "We will continue to give full play to our policy advantages to allow more international innovative medical achievements to benefit Chinese patients, especially those with rare diseases."
6. Patient Story: A Family Regaining Hope
Xiaojie (pseudonym), a 9-year-old child from Hunan, is one of the beneficiaries of Varmololong. His mother, Ms. Wang, shared: "After three months of treatment, Xiaojie's muscle strength has been significantly enhanced, and now he can go up and down the stairs by himself. This seems like a miracle to us."
Similar stories continue to be staged in Lecheng. As treatment continues, more and more children with DMD are expected to achieve better quality of life and development prospects.
The successful application of Varmololong in Lecheng not only brings hope to patients with DMD, but also demonstrates the huge potential of Chinese medical innovation. With policy support, more "life-saving drugs" will accelerate their entry into China and change the fate of patients with rare diseases.
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