Bionews Staff, writers and editors —

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…

Photo courtesy of Ben Mattlin Day 31 of 31 This is Ben Mattlin’s (@benmattlin) story: The disability rights movement in the U.S. is known for many achievements — the Individuals with Disabilities Education Act, blocking buses to demand accessible transit, the Capitol Crawl. But its crowning accomplishment to…

The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both in-person and online. “Supporting charitable causes and organizations are a long-standing tradition of the TCS New York City…

Photo courtesy of Melissa Milinovich Day 30 of 31 This is Claudia Milinovich’s story: My mom is a normal mom. She taught me to walk and talk — stuff any normal parent would do. Yeah, she can’t walk because she has SMA, but that hasn’t stopped her from being…

Photo courtesy of Keith Schaper Day 29 of 31 This is Keith Schaper’s story: Our son, Oliver, was officially diagnosed with SMA type 2 at 14 months old on March 7, 2017. We discovered Cure SMA and swiftly got Oliver, now 5, registered. First came an overnight…

Photo courtesy of Autumn   Day 28 of 31 This is Autumn’s (@itsss.autumn.here) story: Hi! I’m Autumn. I was diagnosed with SMA at 13 months old. SMA is definitely a journey, an adventure: the machines to help me, the surgeries, and learning how to navigate a world that wasn’t…

Photo courtesy of Rachel O’Hagan Day 27 of 31 This is Rachel O’Hagan’s (@bravelikebeau) story: I am a special education teacher and have held the hands of several parents as they navigated a new diagnosis for their child. But nothing prepares you for when it is your own.

Photo courtesy of Carissa Decelles Day 26 of 31 This is Carissa Decelles’ (@rheelgirl) story: Hello! My name is Carissa, and I am a 30-year-old, award-winning art director and graphic designer from eastern Connecticut. I also happen to have SMA type 2. SMA has never been a…

Photo courtesy of Edmund Lim Day 25 of 31 This is Edmund Lim’s story: There were times, after my younger son was diagnosed with SMA type 1, that I was on the verge of resigning his life to fate, feeling futile after years of trying to find a…