When Leonidas Boyne was born, he seemed—for all intents and purposes—to be a healthy, happy child. At five months old, he is still a bubbly, happy baby with a smile…
High-risk neuroblastoma can be difficult to treat in children. Some research suggests that between 40-50% of children with high-risk neuroblastoma relapse following treatment, causing poor outcomes. When Flora Gentleman was…
It has historically been difficult to encourage the development of therapies for patients with rare diseases. To overcome this, the Orphan Drug Act was passed in 1983. This act…
On January 7th, the Jacksonville Jaguars and Tennessee Titans faced off on the football field. With just 2:51 left in the game, Josh Allen returned a fumble for a touchdown,…
According to a story from empr.com, the Oncologic Drugs Advisory Committee, part of the US Food and Drug Administration (FDA), recently voted 16 to 0 against the investigational drug omburtamab…
In 2019, Rian and Mike Fee celebrated the birth of their son Anderson, who they call Andy. Since birth, Andy has always been such a light in the lives…
In September 2020, Amelia Bradford turned eight months old. Her family loved their fun, wiggly, and happy baby. But then Amelia’s mother Danielle noticed something concerning: a facial bruise around…
Historically, both osteosarcoma and neuroblastoma have been difficult to treat. Therefore, new treatment options are needed for these cancers, which predominantly affect children and teenagers. According to Medical XPress, a…
3-year-old Ruby Ann Grimes is bubbly, happy, and loving. But each and every day, Ruby faces an ongoing battle: Opsoclonus-Myoclonus syndrome (OMS). Last January (2020), Ruby was first diagnosed with…
If I told you that tiny bubbles could be used to more effectively treat cancer, would you believe me? Well, in the case of neuroblastoma, that might be correct. According…
For the first time, a research study has discovered a genetic marker related to high-risk neuroblastoma. According to Medical XPress, researchers determined that anaplastic lymphoma kinase (ALK) gene mutations and…
February 2nd is recognized annually as Neuroblastoma Awareness Day, which is intended to help spread knowledge and visibility about neuroblastoma, a rare cancer that mostly affects children, among the general…
Kuur Therapeutics has recently released interim data on their CAR-NKT cell therapy platform, which was created for the treatment of hematological and solid malignancies. This data comes from two phase…
The FDA has recently approved DANYELZA for the treatment of neuroblastoma in pediatric patients over the age of one and adult patients affected by refractory or relapsed neuroblastoma. This approval…
Recently, scientists and researchers from the UCL Great Ormond Street Institute for Child Health (GOS ICH) and the UCL Cancer Institute worked to create an innovative solution for patients with cancerous…
According to a story from BioSpace, the radiopharmaceutical company Clarity Pharmaceuticals, Inc. has announced recently that its experimental diagnostic 64Cu-SARTATE™ has earned Rare Pediatric Disease designation from the US Food…
According to a story from BioSpace, the radiopharmaceutical company Clarity Pharmaceuticals has recently announced that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to its…
.jpg)
The biopharmaceutical company Cellectar Biosciences recently announced that the FDA granted its lead phospholipid drug conjugate, CLR 131, Orphan Drug Designation(ODD) for treatment of lymphoplasmacytic lymphoma. ODD is granted by…
Robyn Smyth is a 14-year-old girl from Ireland who was diagnosed with neuroblastoma when she was three. Since then she's undergone countless rounds of chemotherapy, investigational treatments, a treasured period…
According to a story from news-medical.net, a research team affiliated with Princeton University has been hard at work developing a unique tool that utilizes machine learning in order to help…
Rare disease isn't as rare as it sounds. Ten percent of Americans have a rare disease-- and there are almost 7,000 known rare diseases. A rare disease, also known as…
According to a story from Oncology News, a team of researchers affiliated with UNSW Sydney have made a discovery that could have major implications for the rare cancer neuroblastoma, a…
According to a story from falmouthpacket.co.uk, couple Geoff and Judie Davis were devastated when their two year old boy Mikey died after being diagnosed with neuroblastoma. That was more than…
According to a story from the Derby Telegraph, four-year-old Audrina Hatton-Wright was diagnosed with a rare cancer called neuroblastoma about a year ago. This cancer mostly affects children. Doctors initially…
According to a report from ITV, a five-year-old boy from southern England has made the trip across the Bay of Biscay to Barcelona, Spain, where he is receiving an experimental…
© Copyright Patient Worthy
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.
