News & Publications

Brief Introduction

In year 2016, Biogen and Ionis Pharmaceuticals announced the new drug, Spinraza (Nusinersen), to be the first approved medicine in treating SMA and gaining access to the market. SMA patients face difficulties in controlling motor neuron and muscles. If Spinraza is injected into the spine, patients' reserved genes can produce more SMN protein, which helps improving one's mobility. However, the medicine is not capable in repairing the genes. Hence, the condition might be worsened when medication is stopped.

(If you want to know more about the news on new drugs, please click here to learn more)

References:

  1. 什麼是SMA

  2. 明報:3童試新藥有改善一人可自行走10步

  3. ​香港01:脊髓肌肉萎縮症新藥1年費用6百萬元 家屬:我只想孩子健康快樂

  4. News-Medical: 糧食與藥物管理局審批第一種藥物對待子項和成人與脊髓肌肉萎縮

August 2016 Medical Conference

"New Drugs" Q&A

The questions in "New Drugs Q&A" are raised out by SMA members in the Medical Conference, and answered by Prof. John R. Bach and Prof. Kathryn J Swoboda. The answers do not represent the viewpoint of FSMA.        

                                                                       

Question 1: When can Hong Kong SMA kids try the medicine?

Question 2: When is the estimated time for the introduction and popularization of drug?

Question 3: Besides drugs, are there any other treatment plans?

 

Question 4: Along with the increase in age, SMA patients' physical conditions will deteriorate. The function of the drug is to make the conditions better, allow patients to become normal people, or does it has other effects?

 

Question 5: What is the newest drug treatment for SMA?

 

Question 6: How long is the average life expectancy of SMA patients?

Question 7: What is the percentage of inheriting SMA to the next generation?

 

Question 8: How to effectively maintain the physical functioning?

 

Question 9: Some SMA children are using ventilators for breathing. Will the future medical treatment benefit them in no longer using the machines?

 

Question 10: Is the current technology certain in improving the SMA conditions?

 

Question 11: Why Hong Kong does not have a large-scale drug testing?

 

Question 12: What is the result after the third trial of SMNRX drug? Will there be fourth and fifth stages of trial? How could I join the drug testing? Will my four to five-year-old child be eligible for the testing?

Below are professionals' opinions:

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Email/電郵 :info@fsma.org.hk

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© 2017 by Families of SMA (Spinal Muscular Atrophy) Charitable Trust.