According to a story from Angelman Syndrome news, Angelman syndrome was recently given its own disease unique disease code: Q93.51. The new code will help make researching and tracking the…
According to a story from IRB Barcelona, a recent conference has brought together researchers from around the world that are dedicated to the study of the cell cycle and two…
According to a story from Business Wire, the company Alexion Pharmaceuticals recently announced that it has submitted a Biologics License Application (BLA) to the FDA for the approval of one…
According to a story from CheckOrphan, Express Scripts has prepared a Rare Conditions Care Value (RCCV) program. This program is designed to help make the diagnosis and treatment process for…
According to a story from The Spectator, Madeline Pitts, who has two forms of terminal cancer, will have June 9th be dubbed "Maddie Day" in her honor. She is thirteen…
According to a story from apnews.com, Mike Coble, a senior at Byron High School in southeastern Minnesota, was recently diagnosed with Guillain-Barre syndrome. Mike is on three different sports teams…
According to a story from tdn.com, the radio host Jamie Dupree will soon return to work after nearly a two year absence. However, he will not be doing any announcing…
On June 28th, 2018, the National Society for Phenylketonuria (NSPKU) is holding a special event that is meant to challenge Members of Parliament (MPs) across the UK. This challenge is…
According to a story from globenewswire.com, the corporation Retrotope recently announced the initiation of single-patient, expanded access trials of its experimental product RT001. The patients involved have familial encephalopathy with neuroserpin…
According to a story from Newswise, a team of scientists and researchers have recently completed research that managed to reveal the genetic nature of the unusual rare disease neuromyelitis optica.…
According to a story from UK Healthcare, a team of researchers and scientists at UK Healthcare is preparing to start a clinical trial that will test a new therapy aimed…
According to a story from BioPortfolio, the corporation Herantis Pharma Plc recently announced that its Phase 2 clinical trial measuring the effectiveness of Lymfactin, a gene therapy, in the treating…
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 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 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 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…
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…
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…
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…
PatientWorthy will be offering a pair of tickets to the Alagille Syndrome Alliance's (ALGSA) First Annual Gala. The tickets will be available for patients or caregivers in the San Diego…
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