Blog

Roche announces global pre-approval access/ compassionate use (PAA/CU) plans for Risdiplam On 14 January 2020, Roche announced a pre-approval access/ compassionate use programme for its investigational drug Risdiplam to their affiliates globally and to patient advocate groups in Europe and UK. According to the community letter shared by SMA Europei, this program will initially be for Type 1 SMA patients but will expand to Type 2 SMA patients upon filing for market authorisation. To our knowledge, there is no communication

SPINAL muscular atrophy (SMA) is a rare disease that used to lead to no other outcome but premature death. And before that 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; SMA was known as a major cause of the death of babies under that age. However, with advancements in science and medicine, the outcome for SMA patients has

The Phar-East conference held in March 2019 is a leading pharma and biotech conference for Asia.Being an industry conference, it was interesting to be invited back again to participate in a panel discussion, and this time to also present on the opportunities and challenges of a stakeholder engagement journey from a patient advocate perspective.  I spent the large part of the conference attending various talks on immunotherapy, clinical trials, pharma 4.0, market access and pricing, regulatory affairs; and one topic

My birthday wish – to play Chinese chess. Yoa Wen, 15 years old will turn 16 on the 20 March. He has SMA and is currently staying at home. Yao Wen is a happy, positive and brilliant boy. However, he is not able to go to school in the last few years. He looks forward to have friends and someone to play Chinese chess with him as it his favourite game. As he can only move his right thumb, he

On Aug 8, members of the public were invited to contribute their ideas and proposals for the preparation of Budget 2020 which will be tabled on Oct 11, through a special website. Finance Minister Lim Guan Eng said this would enable all segments of society to submit views in line with the government’s efforts to build the nation and prosperity of the people. We were extremely thrilled and grateful with the opportunity given as it is so rare or has

Branden Lim was testing out a prototype wheelchair when Malaysiakini arrived at his home in Damansara Jaya for an interview.   “Wheeee,” the seven-year-old said with delight as he navigated his jerky wheelchair using an iPad.   Branden has a severe form of spinal muscular atrophy, which leads to loss of motor neurons and progressive muscle degeneration.   His parents, Edmund Lim and Yap Sook Yee, explained that the wheelchair is a prototype for a line of smarter and more responsive