Spinal Muscular Atrophy (SMA) is a rare disease and used to lead to no other outcome but premature death.
Often before death, children would suffer dreadful deterioration of the ability to move, eat and breathe. In a majority of cases, that traumatic end, used to come before two years of age, and SMA is known as an important cause of death of babies under that age.
However, with the advancement of medicine and science, the outcome for anyone with SMA can dramatically change. Today, there are two drug therapies that can stop this disease and save countless lives. Lives which carry a huge amount of potential, looking at the achievements of those with SMA who survive and excel in the arts, profession, entrepreneurship, academia and many other fields.
Whilst there is no access (yet) to life saving drug therapies for SMA in Malaysia, the community is encouraged and regaining hope for a number of reasons.
First, the real possibility of a third drug therapy called Risdiplam. On 25 November 2019, Roche announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) and granted Priority Review for Risdiplam. The FDA is expected to make a decision on approval by 24 May 2020.
Second, our government appears receptive to not shirking responsibility for the lives of the SMA community; one baby is born every week with SMA. SMA has been addressed in Parliament more than once, and recently the Deputy Health Minister YB Dr Lee Boon Chye responded to Batu Gajah MP Sivakumar Varatharaju Naidu’s question on the number of SMA patients treated with orphan drugs by saying “With two treatments now available worldwide, the drug makers have failed to register them here, hence no patient has been treated with the life-saving drug”. The Deputy Minister of Health added that if the application is submitted, the MOH will give due consideration. (Hansard DR 20.11.2019 pg8)
It’s time for the drug makers to step up, to play their part and show their commitment.
Third, the Deputy Health Minister YB Dr Lee Boon Chye explained the plans for a National Framework for Rare Disease to define rare disease in Malaysia, including to craft policies and strategies to obtain orphan drugs and products in Malaysia, amongst other equally important initiatives. (Hansard DR 20.11.2019 pg8)
Fourth, to help drive forward the National Framework for Rare Diseases, the MOH has facilitated the engagement of patient advocacy groups who are able to contribute to the work that is needed. We applaud the spirit of inclusion, and hope that this will continue to strengthen in this New Malaysia defined by equity and broad representation.
Whilst the recent budget for a “Rare Disease” fund has not been increased, we continue to advocate for its greater expansion, inclusiveness, and equity. We hope that with the steps being taken, live saving treatment for those with SMA and other rare diseases will become a reality in year 2020.
It’s early days and we are cautiously optimistic, but hopeful.
Last, but not the least, we are grateful to hear from MPs from both ruling and opposition parties, acknowledging the need to treat SMA. Not only the better detection and response to SMA, but also prevention. Thank you YB Datin Mastura Binti Mohd Yazid and YB Sivakumar Varatharaju Naidu.
Our children with SMA look to our elected representatives to ensure that all Malaysians are valued, cared for and supported. Not just their future but their lives depend on this.
This letter is written by Edmund Lim, father of child with SMA and founder of Persatuan WeCareJourney and endorsed by the following:
- Ainaa Farhanah Binti Amali (adult with SMA)
- Aishah Roose (adult with SMA)
- Amrun Shah (father of a child with SMA)
- Azlin Mastura Binti Ismail (mother of a child with SMA)
- Chai Cheng Sheng (Advisor to Persatuan WeCareJourney)
- Chan Wei Kuan (mother of a child with SMA)
- Chin Siow Veun
- Datin PH Wong, Project Director Childline Foundation
- Datin Seri Sweelin Jayasiri
- Dato’ Dr Amar-Singh HSS, Senior Consultant Paediatrician
- Datuk Seri J.Jayasiri
- Dayana Sheda Al-Azmi (mother of a child with SMA)
- Dr Fahisham Taib, Paediatrician HUSM
- Khairul Azwan Bin Ismail Anuar (father of a child with SMA)
- Lee Pui Ling (mother of a child with SMA)
- Lee Yi Ling (adult with SMA)
- Nik Nur Aida
- Norhafizah bt Sholihin (mother of a child with SMA)
- Padli Ahmad (father of a child with SMA)
- Prof Dr Sharaf Ibrahim, Orthopaedics (ex-HUKM)
- San Yuen Wah
- Shaharatul Azmh binti Shamsudin (mother of a child with SMA, Vice President Persatuan WeCareJourney)
- Shobanahera Raj (mother of a child with SMA)
- Tan Sau Eng (father of a child with SMA)
- Yap Poh Choo
- Yap Sook Yee (mother of a child with SMA, Co- Founder of Persatuan WeCareJourney)
- Yoon Sook-Yee
- This is the personal opinion of the writer or publication and does not necessarily represent the views of CodeBlue.