As sourced from the Washington Post, COVID-19 coverage has been spreading the news: people with many disabilities are scared, and we need to take care of them. For many, the…
A recent University of Sheffield news release explores findings which highlight a potential cause of leukodystrophies: immune cells. This group of rare childhood neurological disorders impacts the myelin sheath, the…
This week I heard from a grandfather who had been wanting to take his grandson fishing. The grandson had always been too busy and had far too many organized activities.…
Sean Boyle is a PhD candidate at Emory University. He just signed up to participate in a trial for a coronavirus vaccine when he doesn't know the risks. That's because…
The month of May is recognized as Myositis Awareness Month. The ultimate goal is to help spread awareness and educate both the general public and the healthcare field about myositis,…
Lynparaza is back in the news again, but this time, according to Cure Today, the FDA approved the drug in combination with Avastin for advanced ovarian cancer to prevent progression…
By Natalie Homan from In The Cloud Copy It’s estimated that about 3.4 million Americans have epilepsy, one of many conditions that can cause seizures. Seizures occur when there is…
A new study published in Blood has found different genetic variants of myelodysplastic syndromes (MDS). The most common of these is called SF3B1, which occurs in around 1 in every 5 patients. It…
Axcella, a biotechnology company based out of Cambridge, Massachusetts, has released the results of AXA1125-003. According to Biotech 365, this study showed positive results for those with non-alcoholic fatty liver…
Chive Charities is a 501(c)3 non-profit organization committed to providing patient advocacy and support. They have raised money for a young girl with a mutated CAMK2B gene to receive intensive physical therapy at…
by Danielle Bradshaw from In The Cloud Copy A year-long study has shown that Tracleer greatly reduces the skin-related symptoms of scleroderma. The results also showed that although it helped…
by Jodee Redmond from In the Cloud Copy Emma Larson’s parents were not particularly concerned when their daughter wasn’t standing or walking by her first birthday. They figured that plenty of children had not reached…
May is recognized annually as Cystic Fibrosis (CF) Awareness Month. Each year, this rare disease community comes together in order to help spread awareness about cystic fibrosis among the general…
Bronchiectasis News covers a potentially beneficial treatment for patients with bronchiectasis: bubble-positive expiratory pressure, or Bubble-PEP. These devices help clear mucus from the lungs. Although other forms of airway clearance…
A total of one hundred vaccines are being developed by researchers, drug manufacturers, and governments worldwide in an effort to stem the tide of the COVID-19 virus. Reuters Newswire reports…
By Danielle Bradshaw from In The Cloud Copy Seventeen-year-old Marissa, from Clinton Township, Michigan, might be what most would consider a model student. She earns good grades and she really enjoys…
According to a story from Xconomy, the drug company PTC Therapeutics has come to an agreement that will allow it to acquire Censa Pharmaceuticals and its lead product candidate. This…
ProMIS Neurosciences is a biotechnology company that works specifically with antibody treatments, and they have recently developed intrabodies for the treatment of amyotrophic lateral sclerosis (ALS). This same therapy has…
It is acknowledged in the medical community that clumps of misfolded tau proteins and amyloid-β are markers of Alzheimer’s disease. The evidence presented in recent article in Nature, shows that…
According to UIC Today, Jessica Lechuga became immersed within the rare disease community when she was still a teenager. That's because, at age 17, Jessica gave birth to her daughter…
According to a recent article in BioMed Central, an international task force formed in 2018 created guidelines for the treatment of juvenile idiopathic arthritis (JIA). Treatment targets were identified…
Hello! We hope that within all this chaos, you're still able to enjoy some parts of spring. Today, we have a story about kids with disabilities who are affected by…
Merck and AstraZeneca recently announced that Lynparza improved overall survival in the lives of males with advanced prostate cancer who had been treated previously. The drug out-performed two other antiandrogen…
According to a recent announcement by AstraZeneca, the U.S. FDA granted its approval for Koselugo (selumetinib). This is the first drug to be approved for the treatment of children…
Continued From Part One written by Payel Bhattacharya Pharmaceutical science has medications to control the pain. I was initially put on medicine but it mostly didn’t contain the pain. I…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
