My 14-year-old daughter woke me up at 4:45 a.m. “Do you hear that?” “Hear what?” I asked groggily. “You don’t hear anything?” Panic rose in Sara’s voice. “No.” I sat up. “You don’t hear that?” she said, just after I too heard a high-pitched chirp. “Yes,” I said. “It sounds…
Like many couples, Joe and Linda Lacroix of Milton, Vermont, spent much of their lives working, raising families, maintaining their home, and planning for the future. Unlike most, however, there was always a dark cloud looming on their horizon — a gene for Huntington’s disease carried by Joe’s mother.
The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…
After three weeks of my husband, Todd, being cloistered in our home with a cold, we ventured out Saturday for Michigan Tech’s last home hockey game of the season. Our Huskies took on their archrival, the Northern Michigan Wildcats. Games between the two teams usually sell out because…
In recognition of Rare Disease Day Feb. 29, Bionews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the #WhatMakesMeRare campaign was aimed at uplifting people with rare diseases by encouraging them to share their stories and perspectives. The…
Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, and aims to supports work by researchers and clinicians into a better understanding of and treatments for rare diseases, as well…
Astrocytes — cells of the nervous system cell that provide support to neurons — were seen to help protect motor neurons from damage caused by toxic protein clumps in a cell model of amyotrophic lateral sclerosis (ALS). The study reporting this finding, “Distinct responses of neurons…
Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those looking to begin the complex process in its Feb. 20 webinar. William Whitman…
The other day, I was chatting with a friend about Rare Disease Day. Wait, what? You don’t know about Rare Disease Day? Well, don’t feel bad. Last year I missed it, thinking it was just another ho-hum awareness event. But I’ve learned the value of events such as this…
Biotech company EnClear Therapies has announced $10 million in financing to help advance its proprietary platform for stopping the progression of neurodegenerative disorders, including amyotrophic lateral sclerosis (ALS). The investments comes from a Series A financing round, which is part of an early development stage for start-up…
