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Rare Diseases

Adult SMA Patients Left in the Lurch with Delayed Treatment Access

women holding placards curesma
women holding placards curesma
Canadian adults are still progressing as they wait for access to treatment. Photo taken three years ago.

Mental health concerns are rampant more than ever among adult patients with Spinal Muscular Atrophy (SMA). Cure SMA Canada is advocating for treatment access for adult patients with the progressive disease.

The information highway is fast and furious in terms of incoming research data for Spinal Muscular Atrophy (SMA), and the results have been astounding for patient benefit. To paint a picture, Cure SMA Canada sent out newly diagnosed patient care packages with the knowledge that several packages would be followed up with an end-of-life package within months or a year. After the first treatment became available in Canada, those end-of-life packages dropped to almost none.

Fast forward three years, that information highway ran parallel to the road of advocacy — filled with roadblocks and potholes. The fight for access to treatment is daunting, exhausting, and filled with frustration and heartache, however, it is one that we have no choice to travel because lives are saved by the innovative treatments that have been developed.

A group of people have watched this all unfold — the same group that has lived a life of watching their disease progress over the years. They have experienced a loss of function with walking, rolling over in bed, brushing their hair or maybe even swallowing, all the while their mental capacity has remained completely intact. Most adults have grown up with the knowledge that there’s no treatment for SMA — they have raised funds and awareness, watched clinical trials move through their phases, waiting and hoping.

Decisions affect more than just the patient, it affects the family unit, as well as the extended family, friends and community.

Imagine the roller coaster of emotions for an adult patient who is still experiencing the ramifications of living without treatment access, as three treatments move through the gamut of approval. There are the physical and emotional consequences of living with a progressive disease, and then far more crushing is the knowledge that there is an effective treatment for adult SMA patients that is available in other jurisdictions. To say that this has a severe emotional impact would be an understatement. Mental health concerns are rampant more than ever among adult SMA patients.

Patients with access to treatment have reported experiencing more stamina and strength, and most importantly disease stabilization. They are able to hold their jobs, hold their children, and even hold their toothbrushes. All three are powerful abilities, imagine losing one of those.  Imagine losing one and gaining it back. Now imagine watching someone else gain it back and wonder when your turn is and worrying that your turn will come too late.

This is where we are at now, urging government bodies to understand the value of adult SMA patients. Cure SMA Canada feels that they should have been included in the conversation right from the start. Now with even stronger evidence supporting their access, there is no excuse to leave them out. The clock keeps ticking and every day without treatment is a day of progression for adult patients who are still waiting.

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