Every day, people around the world learn more about Huntington’s disease (HD). We know so much today, but we will know more tomorrow, and this knowledge will continue to exponentially grow. Researchers, scientists, clinicians, patients, caregivers, and advocates all have knowledge to gain. If you ever think you know it…
Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…
In honor of Valentine’s Day last year, I wrote my wife a love letter. I’ve decided this will be a tradition, so following is another one. Dear Jill, Last February, COVID-19 was on the cusp of steamrolling the world. Little did we know that in March, you would become…
The British Psychological Society has released evidence-based recommendations for health professionals managing the psychological health of people with Huntington’s disease and other neurodegenerative disorders. Its document noted the lack of research on psychological interventions and the poor access to specialized mental health services for these patient groups, highlighting an urgent…
Note: This story was updated Feb. 9, 2021, to clarify details surrounding the grant Chronic Care Collaborative received from Adira. Greg Smiley’s world changed abruptly in 2013. He was racing down a mountain road on an outback cycling trip in South Africa, when he hit an obstruction in the road…
While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention. Those were the findings of the…
Late last year, my wife, Jill, and I challenged ourselves to a 21-day negativity detox. With so much gloom and cynicism in the world, we decided to work on being positive. Jill has Huntington’s disease, and stress makes her symptoms worse. We kept track by placing a dollar…
Higher-than-normal levels of mu-opioid receptor 1 (MOR1) in the striatum — a brain region particularly affected in Huntington’s disease — may contribute to both early psychiatric symptoms and late motor difficulties seen in Huntington’s patients, according to a study in a mouse model of the disease. The data also…
There is a robust pipeline of potential therapies for Huntington’s disease (HD), which I’ve discussed in previous columns. All of the current and future clinical trials provide real hope for this community. However, people typically aren’t allowed to participate in two clinical trials at the same time, and…
The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…
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