Roche Compassionate Use Treatment for Palden with SMA Type 2

Roche Compassionate Use Treatment for Palden with SMA Type 2

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Started
Petition to
Roche

Why this petition matters

Started by Namgay Zam

Dear Mr. Schwan (CEO),

I write this petition on behalf of 13-year-old Palden Dakini Dorji, the only Bhutanese with SMA Type 2, and to request Roche to consider her for the compassionate use program.

Forget an individual from Bhutan being able to afford this treatment, even the Health Ministry cannot afford it for one Bhutanese. I am told that the cost for annual medical access amounts to about USD 340,000 which is about Nu. 25M - more than a lifetime’s earning of a Bhutanese civil servant at the executive level! Even a year’s access is impossibly expensive for a Bhutanese; lifelong access is a dream! But we know Roche can make this dream come true for Palden.

Bhutan has seen only two SMA cases till date. Palden’s is the first case. The country neither has the expertise nor facilities to help Palden live a full life despite her deserving it. She is a bright, happy, and optimistic schoolgoing little girl, who gives her best everyday in spite of the increasing challenges posed by her condition.

Palden weighs only 13.5 kilos. At the time of this petition, she can barely open her mouth to eat or talk, and has no feeding tube even. You sir, are most aware of this rare condition and know how urgent immediate care for someone like her is.

Palden’s only hope for life with less pain is Roche’s compassionate use treatment. So this is all of us from Bhutan and elsewhere beseeching Roche to help Palden because we believe Roche can and will help her.

Thank you. 

There are three main treatments in the world for SMA that have all started to become available only in the last two years after decades of fundraising and science trying to find treatment.
Bhutan does not have have access to all three treatments.

Of the three treatments available, Zolgensma is only for babies, Spinraza requires hospital admission every four months to receive a lumbar puncture, and Risdiplam (brand name Evrysdi) can be taken orally every day at home.

Due to Paldens age, the lack of hospital facilities and respiratory machines such as bi pap, and Palden's scoliosis (lumbar puncture is not possible), the only possible treatment is Risdiplam. This is produced by Roche. 

 

13,058 have signed. Let’s get to 15,000!