The experimental gene therapy LentiGlobin for the treatment of severe sickle cell disease is being investigated in an ongoing Phase 1 clinical trial. Bluebird Bio, the company developing the therapy,…
According to a story from the Ottawa Sun, several hundred people participated in the annual ALS Walk on June 9th, in which people walk down the Sir John A. MacDonald…
According to a story from globenewswire.com, the biopharmaceutical company Idera Pharmaceuticals recently released the data from its Phase 2 trial for IMO-8400, an investigational product in development for the treatment…
According to a story from PR Newswire, the biotechnology company REGENXBIO recently announced that the U.S. Food and Drug Administration (FDA) has granted its experimental gene therapy product RGX-111 Fast…
According to Bright Surf, a recent study has led researchers to believe that children with neuroblastoma are at a higher risk of developing long-term psychological impairment. Neuroblastoma is a rare…
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…
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…
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…
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,…
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…
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…
Happy Father's Day weekend! While Father's Day can be complicated in a rare family, rare parents deserve a round of applause. This week we have a Father's Day tribute from…
According to a story from pm360online.com, the speciality pharmaceutical company Chiesi USA and the biotech company Holostem Terapie Avanzate S.r.l. of Italy recently announced that the U.S. FDA recently granted orphan…
Newron Pharmaceuticals S.p.A (Newron) is a biopharmaceutical company specializing in developing inventive therapeutic options. They focus primarily on conditions related to the nervous system. According to bioportfolio.com, Newron announced on…
According to BioPortfolio, there was recently an announcement about preclinical data for SYNB1618, which is a Synthetic Biotic treatment that is being assessed right now in a Phase 1/2a clinical trial in…
According to EurekAlert!, a study at Virginia Commonwealth University has potentially pinpointed the so called "Achilles heal" of a certain type of deadly brain cancer: glioblastoma multiforme. Glioblastoma, or glioblastoma…
According to a story from med.miami.edu, a team of researchers working at the Sylvester Comprehensive Cancer Center, which is part of the University of Miami School of Medicine, made a…
According to Globe Newswire, the United States Food and Drug Administration (US FDA) designated a new orphan drug for the treatment of multiple myeloma on May 9th, 2018. The drug…
Lawmakers in the U.S. state of North Carolina have expanded the state’s newborn screening testing to include three more conditions. These are Pompe disease, Mucopolysaccharidosis type 1 (MPS 1), and…
According to Charcot-Marie-Tooth News, a Chinese family of five who all have the same type of Charcot-Marie-Tooth (CMT) disease share something else in common as well. Not only do they…
Darwin Ecosystem typically is a name heard in analytic circles. The company specializes in machine-learning and pattern recognition. Now, the company is using their artificial intelligence know-how to help give…
According to Medical Xpress, scientists have pinpointed how a potentially new treatment effectively targets spinal muscular atrophy (SMA). The results from the research could lead to further development in SMA…
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