Cure SMA Featured in Global Genes’ Patient Voice Project


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Global Genes, a leading rare disease patient advocacy organization, recently published a new toolkit titled “From Molecules to Medicines: How Patients Can Share Their Voices Through the Drug Development Process” to address the challenges of patient participation in drug development.

According to a press release, the toolkit was the result of a collaboration between Global Genes, advocacy leaders and governmental agencies. Cure SMA also collaborated in the project, with its work used as case studies in five different ways: to encourage research; to fund research; to provide data; to participate in trials; and to participate in committees.

Drug development is changing every day, with increasing patient participation – including their families and caregivers – accounting for one of the most fundamental changes the industry has adapted to so far. Traditionally, pharmaceuticals and regulatory agencies included patient participation at specific points during drug development, most commonly by the time a new drug was close to being approved.

But the knowledge of patients and caregivers was not included as a standard measure at certain key points such as targeting early research, assessing the benefits and risks of novel therapies, developing regulatory guidelines, or designing clinical trials.

But the situation is changing. Global Genes’ toolkits, which are part of a series on drug development, hope to highlight how patients who suffer from SMA and from other conditions are using their voices to guide conversations, research, and drug development, and how these voices are yielding considerable advances in their communities.

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Cure SMA has awarded a $90,000 grant to Antoine Cléry, Ph.D., a researcher at ETH Zurich, for his work on spinal muscular atrophy titled “Novel approaches against spinal muscular atrophy by targeting splicing regulators.

The award is part of an $890,000 new basic research fund recently announced by Cure SMA – a strategic step in its comprehensive research model to investigate the causes of SMA and identify the best strategies for eliminating the disease.