Will there ever be a cure for SMA?
There is no cure for SMA. Treatment consists of managing the symptoms and preventing complications. The U.S. Food and Drug Administration has approved the drug nusinersen (Spinraza ™) to treat children and adults with spinal muscular atrophy.
What is CURE SMA?
Cure SMA is a voluntary-driven, non-profit organization dedicated solely to eradicating SMA by promoting and supporting research, helping families cope through informational programs and support, and educating the public and the medical community about SMA. Spinal muscular atrophy is a motor neuron disease.
Is there a cure for SMA type 1?
Currently, no cure exists for SMA, but three disease-modifying therapies have become available since 2016 that have the potential to slow or even prevent progression of the main types of the disease.
Can Crispr Cure SMA?
Gene editing technologies, such as the CRISPR‐Cas9 system, may provide yet another way to treat SMA in the future. CRISPR‐Cas9 uses an engineered enzyme to make precise modifications to DNA sequences. Laboratory experiments have shown that CRISPR‐Cas9 can correct pathogenetic mutations at the embryonic stage.
Why is Zolgensma not a cure?
However, zolgensma is not a cure and this is due to several reasons: Although zolgenmsa can increase SMN protein in motor neurons, for it to be a cure, it must do so in most, if not all, motor neurons and all other cells which have insufficient SMN. Unfortunately this is unlikely to occur.
How long can you live with SMA?
Life expectancy Most children with type 1 SMA will only live a few years. However, people who’ve been treated with new SMA drugs have seen promising improvements in their quality of life — and life expectancy. Children with other types of SMA can survive long into adulthood and live healthy, fulfilling lives.
Why can’t spinal atrophy heal?
Treating SMA Due to a mutation in the survival motor neuron gene 1 (SMN1), individuals with spinal muscular atrophy (SMA) don’t produce survival motor neuron (SMN) protein at high enough levels. Without this protein, the motor neuron cells shrink and eventually die.
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