Current Drug Treatments for Neurofibromatosis in Taiwan - Plastic Surgery

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Pharmacological treatment for neurofibromatosis?


Hello Dr.
Lu, I am a patient diagnosed with a rare disease, neurofibromatosis, since birth.
I would like to ask if there are currently any medications available in Taiwan to treat neurofibromatosis.
I came across an article stating that a medication has been developed in the United States for treating neurofibromatosis, but I am unsure if this medication has been introduced in Taiwan.
If it has, could you please let me know which hospital offers this neurofibromatosis medication? I found this information in the following article: https://www.gbimonthly.com/2020/04/66723/

Xiao Feng, 30~39 year old female. Ask Date: 2021/04/26

Dr. Lü Mingchuan reply Plastic Surgery


Hello: After reading this website and researching some information, I found that currently, there is no such medication available in Taiwan.
Even in the United States, the FDA has only approved this product in the past one to two years, and it has recently been made available for neurofibromatosis.
However, there is still hope.

First, seeking treatment abroad is certainly an option.
Additionally, you can inquire with some pharmaceutical companies about becoming a distributor.
The Ministry of Health and Welfare indicates that the prevalence of neurofibromatosis is approximately 1 in 40,000, which means there are likely fewer than a thousand people affected in Taiwan.
Although it is considered a rare disease, it has a relatively higher number of cases compared to other rare diseases.

Moreover, this medication is manufactured by the same company, AstraZeneca, that produces the AZ vaccine.
It is recommended to explore this direction.
The Changhua Hospital of the Ministry of Health and Welfare cares about you.

Reply Date: 2021/04/26

More Info


Neurofibromatosis (NF) is a genetic disorder characterized by the growth of tumors on nerves, skin changes, and other abnormalities. There are three main types of neurofibromatosis: NF1, NF2, and schwannomatosis, with NF1 being the most common. The management and treatment of neurofibromatosis can be complex, as it often involves a multidisciplinary approach due to the variety of symptoms and complications associated with the condition.

As of now, there are no specific drugs that can cure neurofibromatosis; however, there are treatments available that can help manage symptoms and complications. In recent years, targeted therapies have emerged, particularly for NF1. One of the most notable advancements is the approval of a drug called selumetinib (brand name Koselugo), which is designed to treat inoperable plexiform neurofibromas in pediatric patients with NF1. This drug works by inhibiting the MEK pathway, which is often activated in NF tumors. Clinical trials have shown promising results, leading to a reduction in tumor size in some patients.

In Taiwan, the availability of selumetinib and other targeted therapies may vary. It is essential to consult with a healthcare provider who specializes in neurofibromatosis or genetic disorders to determine the most appropriate treatment options. Major hospitals and medical centers in Taiwan, such as National Taiwan University Hospital, Taipei Veterans General Hospital, and Chang Gung Memorial Hospital, often have specialized departments for genetic disorders and may offer access to the latest treatments and clinical trials.

In addition to pharmacological treatments, management of neurofibromatosis often includes regular monitoring and supportive care. This may involve surgical intervention for symptomatic neurofibromas, physical therapy for associated musculoskeletal issues, and psychological support for patients and families coping with the challenges of living with a chronic condition.

If you are interested in the latest treatments or clinical trials, it is advisable to reach out to these hospitals or consult with a specialist in genetic disorders. They can provide information on whether the latest drugs, such as selumetinib, are available in Taiwan and guide you through the process of accessing these treatments.

In summary, while there is no cure for neurofibromatosis, advancements in targeted therapies offer hope for better management of the condition. It is crucial to stay informed about new developments and work closely with healthcare professionals to ensure the best possible care.

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