KUALA LUMPUR: The Health Ministry has been urged to expedite access to a new drug therapy to treat those affected by spinal muscular atrophy (SMA), especially children, said founder of NGO WeCareJourney Yap Sook Yee.
Yee said that SMA is a progressive muscle wasting disease and one of the top genetic killer of babies below the age of two years old.
"Two years ago a new drug therapy which stops SMA called Spinraza was approved by the US Food and Drug Administration (FDA) and has been made available in many countries for children diagnosed with SMA.
"Our SMA community needs the Ministry's support to ensure that children suffering from the illness have access to the new therapy similar to other rare diseases which are already funded by the government," she told a press conference at the Parliament lobby on Monday (Nov 19).
She was accompanied by Petaling Jaya MP Maria Chin Abdullah, who met with Deputy Health Minister Dr Lee Boon Chye to discuss the matter.
Yap also urged the government to consider and include SMA in the rare disease fund allocated under Budget 2019.
"We applaud and appreciate the effort by the Ministry to allocate funds to help those who are most vulnerable with the most unmet needs.
"Our community has been campaigning for the past two years to have this drug made available in the country.
"Evidence exists supporting the effectiveness of this medication. The disease is progressive and terminal, every day counts for these children.
"Children with SMA can grow to become active citizens who can contribute to our country if they are given the chance," she said.
Meanwhile, Maria urged the Health Ministry to consider the suggestions.
"We were informed that a taskforce will be set up to look into it," she said.