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NICE has issued a draft guideline recommending risdiplam (Evrysdi) as part of the Management Access Agreement (MAA) for the treatment of the rare genetic disease spinal muscular atrophy (SMA)
.
It is estimated that approximately 1,500 patients in England will benefit from this drug, which can be taken as a syrup at home
manage
MAA is a special agreement between NHS England, NHS Improvement and Roche, which allows people to use risdiplam for treatment while collecting more data
.
NICE will then decide whether to recommend the NHS routine use of the drug and update the guidelines
MAA is a special agreement between NHS England, NHS Improvement and Roche, which allows people to use risdiplam for treatment while collecting more data
There are several types of SMA.
NICE’s draft guidelines recommend that risdiplam be used for patients who are clinically diagnosed with SMA type 1, 2 or 3, or for patients with pre-symptomatic SMA and the number of surviving motor neurons is 1 to 4 for two months or more.
Of patients
.
Amanda Pritchard, Chief Executive Officer of NHS England, said: “In the past three years, the NHS has revolutionized the care of SMA patients by ensuring access to three innovative therapies—Spinraza, Zolgensma, and now risdiplam.
There are no effective drugs
.
"
Risdiplam is a surviving motor neuron 2 directed RNA splicing modifier
.
Risdiplam solves the root cause of SMA: the decrease in the number of survival motor neuron (SMN) proteins
Infect
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