According to a story from Global Genes, the drug developer SpringWorks Therapeutics announced that the US Food and Drug Administration (FDA) has granted the company Fast Track Designation for its…
According to a story from The Washington Post, Enrique Galvan has been subjected to relentless bullying and rejection ever since he first started school. The reason why? He was diagnosed…
According to a story from Acrofan, the biopharmaceutical company SpringWorks Therapeutics, Inc. has recently announced the that company's experimental product candidate PD-0325901 has earned Fast Track designation from the US…
According to a story from KTVZ, Jill Noe and Whitney Bliesner have always shared a special bond. They are twins, now 34 years old. Despite their closeness, they do share…
A three-year research grant of $11 million has been awarded through the Gilbert Family Foundation in America. According to a recent article in News-Medical Life Sciences, the Foundation was formed…
A new medication called selumetinib has just received Breakthrough Therapy designation by the FDA for pediatric patients diagnosed with neurofibromatosis type 1 (NF1) or inoperable pleciform neurofibromas. This therapy has…
According to a story from EurekAlert!, a collaboration of Sage Bionetworks, The Children's Tumor Foundation (CTF), and the Neurofibromatosis Therapeutic Acceleration Program (NTAP) have announced the initiation of the very…
If you haven't heard of neurofibromatosis (NF), you’re in surprisingly good company. For most of her life, Rachel Mindrup, an artist, illustrator, and Resident Assistant Professor at Creighton University, who…
This weekend Patient Worthy attended RAREfest, an event being held in the UK by the Cambridge Rare Disease Network to raise awareness and promote discussion around rare disease issues. The…
Neurofibromatosis is an extremely rare condition. Worldwide it affects just one in four thousand people. Gail Appelgren and Tim Golumbia are two of the individuals living with the illness and…
According to a story from BioPortfolio, the biopharmaceutical company SpringWorks Therapeutics recently announced that the US Food and Drug Administration (FDA) has given the company's experimental product PD-0325901 Orphan Drug…
The healthcare technology company RDMD was founded by Onno Faber, who has himself been diagnosed with a rare disorder. RDMD, which describes itself as “dedicated to accelerating research” with…
The Live Like Bella Childhood Cancer Foundation was created for Bella Rodriguez-Torres. The Miami born girl had stage four of a rare childhood cancer which took her life at the…
In a study conducted by Ph.D Ludwine Messiaen, the professor of genetics at the University of Alabama at Birmingham, says that there has been progress made to help aid those…
A man in France has been the first person to ever receive two face transplants, reports the BBC. After Jérôme Hamon’s first transplant was rejected he underwent a second transplant…
According to a story from Medical Xpress, the addition of crizotinib with radiation therapy could help reduce hearing loss associated with the treatment in patients with neurofibromatosis II. Crizotinib is…
According to a story from Business Wire, selumetinib, a drug in development for the treatment for neurofibromatosis type 1, was recently granted Orphan Drug Designation by the Food and Drug…
It wasn't the Christmas 18-year-old Alex Leisenring and his family were expecting this year. While they received the white Christmas they had hoped for, they spent the holiday in a…
Although it never should have happened, at least the right course of action was taken! Reggie Bibbs of Houston, Texas was turned away from his Uber driver when he requested…
When she was only 16 months, Poppy was diagnosed with neurofibromatosis (NF). After 60 intense rounds of chemotherapy, she has now lost sight in one eye. NF is a rare…
Michele Holbrook is a runner-- if you spotted her jogging on the street, you wouldn't believe that her body was plagued by more than 20 tumors due to a rare…
Sandra De Santos has thousands of bubble-like tumors on her body due to a rare disease called neurofibromatosis type-1. It's a hard life, but she has spoken out proudly about…
Jessica Brown is gearing up to turn 11 years old on December 20th, and her mother, Jemma, is urging people to show her some birthday love. Jessica suffers from neurofibromatosis,…
Former Jersey Boys star Jeff Leibow, organized his first Neurofibromatosis Hope benefit concert in Las Vegas in 2011. His hope was to make it an annual event that would go…
Our bodies are complex and unpredictable machines. We can try our best to keep them healthy, but sometimes nature does its work, resulting in something we never could have planned…
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