Patient Worthy's partner The MDS Foundation will be hosting a patient forum and and their first ever awareness walk in July! The 5k will be held July 20th and the…
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…
The first patients have been enrolled in two clinical trials of the experimental drug neridronate. The trials are part of the company’s Phase 3 program to evaluate the effects of…
Saturday June 23rd is Dravet syndrome awareness day! Dravet syndrome is a rare genetic dysfunction of the brain (epileptic encephalopathy). It begins in the first year of life in an otherwise…
The U.S. Food and Drug Administration (FDA) has approved the medicine Keytruda for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma, that have relapsed…
Researchers have discovered a new drug that appears to slow cell growth in mantle cell lymphoma. The full article can be read here, at the Bellvitge Biomedical Research Institute (IDIBELL)…
Six-year-old Hope has spent her early years in and out of hospitals for treatment for the neuroblastoma tumors that populate her body- her lungs, stomach, arms, and even her spine…
According to a story from BioPortfolio, the gene therapy company Nightstar Therapeutics recently released an announcement that the FDA issued Regenerative Medicine Advanced Therapy (RMAT) designation for its investigational product…
If you are diagnosed with a serious and rare cancer like acute myeloid leukemia, it is essential that you have access to feedback from leukemia experts. Even if your doctor…
According to Globe Newswire, a pharmaceutical company, Akcea Therapeutics, recently announced their new genetic testing and counseling treatment program for those who may have hereditary ATTR amyloidosis (hATTR amyloidosis). The program,…
Today is World Sickle Cell Day! Established in 2008, this international awareness day is aimed to increase public awareness and an understanding of sickle cell diseases, while educating those who may…
The All of Us program is a huge research project set up by the National Institute of Health (NIH) that aims to collect genetic and health data from a representative…
Based on reports from PRNewswire, there appears to be a rising concern in both the number of cases and the amount of global concern about NASH. NASH stands for Nonalcoholic…
According to a story from PR Newswire, the nonprofit biopharmaceutical company Medicines for Global Health recently announced that the U.S. Food and Drug Administration has approved a drug for the…
According to a story from BioSpace, the biopharmaceutical company AMO Pharma Limited recently announced that the U.S. Food and Drug Administration (FDA) had decided to grant Orphan Drug designation for…
A phase 1/2 study of the drug lumasiran for the treatment of primary hyperoxaluria Type 1 has produced encouraging results, reports Alnylam Pharmaceuticals. The full article can be read here,…
According to UVA Today, a University of Virginia professor, Sarah Kucenas, is studying glia cells in the brain of fish that could potentially help spear the way for the creation of…
According to a story from coloradocancerblogs.org, a recent study demonstrated that data gathered from PET scans could help providers tailor their treatment more precisely in cases of esophageal cancer, resulting…
Promising data from the ongoing Phase I clinical trial Explorer has been announced by Blueprint Medicines Corporation. The trial is designed to evaluate the effects of the experimental drug avapritinib…
According to Neuroscience News, a new study has shown that children with chronic kidney disease, CKD, also display changes in cerebral blood flow that could cause higher risk of cognitive…
According to a story from pm360online.com, the drug development company ProQR Therapeutics N.V. will be partnering with EB Research Partnership (EBRP) and EB Medical Research Foundation (EBMRF) in order to…
According to a story from businesswire.com, the company Prana Biotechnology Ltd recently announced that it is beginning the recruitment process for its Phase 1 clinical trial of its investigational product…
In today's ever-changing - and downright weird - world, you'd be correct to say say (if not over-say) that technology is slowly killing us. But not in this case! Good…
According to a story from CBS News, Clay Emerson, from New Jersey, is hoping to use Father's Day in order to raise awareness about his three year old daughter's rare…
June 17th is worldwide CDKL5 Awareness Day! It's our mission to give a spotlight to the rare diseases that don't often get talked about - and especially on its awareness day!…
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