By Sarah Booth (Herald Sun), March 10, 2024. Read the full article here.
Melbourne, Victoria – In a significant medical breakthrough, researchers have discovered that a drug commonly used to treat vertigo may offer new hope for children suffering from Niemann-Pick type C disease (NPC), a rare genetic disorder often dubbed “childhood Alzheimer’s.”
NPC is a devastating condition that leads to severe neurological decline in children, affecting their movement, speech, and cognitive abilities. The disease is caused by the accumulation of cholesterol and other lipids in the cells, leading to cell dysfunction and death.
The vertigo drug, which has been on the market for years, has shown potential in early-stage research to alleviate some of the neurological symptoms associated with NPC. This discovery is particularly promising because it involves repurposing an existing medication, which could expedite the process of making it available to patients.
“Within the first couple of weeks of starting the drug … we did find some really big, big improvements, which were things that people take for granted every day,” - Mum, Amanda Hodgson
While the research is still in its early stages, the findings represent a significant step forward. The potential to improve the lives of children suffering from NPC and provide relief to their families is a beacon of hope. Further studies and clinical trials will be necessary to confirm the drug’s efficacy and safety for this new use.
A Family’s Journey with NPC
The Hodgson family from Melbourne, Victoria, knows all too well the challenges of living with NPC. Their eighteen-year-old daughter, Autumn, was diagnosed with the disease two years ago – after teachers and parents noticed changes in her speech and movement. Since then, Autumn's parents, have been tirelessly seeking treatments and participating in research studies to help manage her condition.
Despite the grim prognosis, the Hodgsons remain hopeful and proactive, applying for experimental treatments and raising awareness about NPC Research. Their journey highlights the urgent need for effective treatments and the profound impact such a breakthrough could have on families like theirs.
This breakthrough offers a promising new avenue for NPC treatment, and as researchers continue to explore the possibilities, there is renewed hope for those affected by this challenging disease.
Stay tuned for more updates as this exciting research progresses.
Feel free to share your thoughts or any questions you might have about this promising development in the comments below!
To help families like Autumn around Australia, as well as fund vital research for a cure, support the Australian NPC Disease Foundation in its goals of "Research. Cure. Persevere."
Ways to support NP-C in Australia:
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