A New Jersey mother, Raziel Green, 52, was diagnosed with a rare form of ALS in 2017 after years of experiencing symptoms that affected her ability to run and move. Despite initial dismissals by some healthcare providers, she persisted and sought further opinions, eventually being diagnosed with a specific genetic mutation that causes ALS. Learning about a clinical trial for an experimental drug called QALSODY®, Green decided to participate in the trial, administered through lumbar punctures every few weeks. The drug, designed to target the specific genetic mutation she has, has had a significant impact on her disease progression. Within months of starting the medication, Green noticed a marked improvement and has not deteriorated since. The drug works by reducing the harmful effects of the gene mutation, and some patients have even shown signs of improvement. Medical professionals not involved in Green’s care have also praised the drug’s potential, calling it one of the most promising treatments for ALS in recent years.
“In clinical trials for chronic diseases, the true benefits of treatment often emerge over an extended period. According to Purvis, who was not involved in Green’s care, it can be challenging to predict the long-term impact of a newly released drug without following treated patients for an extended duration. Speculation suggests that early administration of the drug, which targets DNA, could potentially offer a curative effect, though conclusive trials have yet to be conducted.
The bond between twin sisters was strengthened upon receiving a rare disease diagnosis, with a commitment to doing their best. Some patients taking QALSODY have reported side effects, with approximately 7% experiencing severe reactions such as myelitis, radiculitis, increased intracranial pressure, and others, as shared by Miller.
Despite facing rare effects like severe headaches, weakness, and sensory loss, Green expressed gratitude for how her medication enabled her to engage in her children’s activities and milestones. Though uncertainties exist regarding the long-term consequences of these therapies, current data suggests their safety over time.
Fradette from Biogen highlighted positive outcomes from the Phase 3 VALOR study, indicating a significant reduction in plasma neurofilament levels among QALSODY-treated participants compared to those receiving a placebo. Green’s mobility is somewhat restricted today, relying on a cane and wheelchair for longer distances; however, her condition has not deteriorated significantly, allowing her to maintain activities she enjoys.
Looking forward, ongoing research aims to slow the progression of neurodegeneration in diseases like ALS, drawing on past lessons to develop safe and effective treatments for the ALS community. With FDA approval, QALSODY is now accessible to individuals with the specific gene mutation, offering hope for improved quality of life and continued independence.
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The medication has provided Green and other patients sharing the same gene with “hope to continue moving forward,” she mentioned. “It also allows my children to undergo testing and receive this medication as a preventive measure,” Green added. “My primary objective was to maintain stability moving forward after undergoing treatment.” Source: Mother with rare ALS praises ‘miracle drug’ halting disease
