Patient Worthy's partner the MDS Foundation is hosting a MDS Patient & Family/Caregiver Forum Baltimore, Maryland in conjunction with the University of Maryland Marlene and Stewart Greenebaum Cancer Center. This…
According to a story from Global Genes, the drug developer Viela Bio recently issued a presentation of the results from a clinical trial that tested the company's investigational product candidate…
According to a story from The Washington Post, a host of different factors ultimately led to the tragic death of Olivia Shea Paregol, a freshman at the University of Maryland.…
Selon un article de BBC News, le père Shakil Malji demande depuis des mois que sa jeune fille Maryam, atteinte d’une maladie rare et mortelle appelé l’atrophie musculaire spinale, puisse…
According to a publication from Express Digest, Britain's National Health Service (NHS) has finally reached an agreement with American biotechnology company Biogen Inc. over the price of Spinraza - a…
According to a story from globenewswire.com, the biopharmaceutical company Poseida Therapeutics, Inc. recently announced that the company's investigational product candidate P-BCMA-101 has been granted Orphan Drug Designation from the US…
According to a story from Kamloops Coffee Talk, Darrin Rein, of Kamloops, British Columbia, was just recently diagnosed with spinocerebellar ataxia, a rare disease that affects mobility and coordination. Despite…
Short Bowel Syndrome Short bowel syndrome (SBS) is a rare condition which occurs when patients have a large portion of their intestine removed. The surgery which leads to this diagnosis…
According to a story from romania-insider.com, there are a total of 26 known cases of hereditary transthyretin mediated (HTTR) amyloidosis known in the country of Romania. However, 20 of these…
The Word "Rare" Unfortunately, many experts in rare disease believe the phrase "rare disease" may be harming the very population it describes. The Director of the National Center for Advancing…
According to a story from BioSpace, a Phase 1/2 clinical trial testing SGT-001, an investigational treatment for Duchenne muscular dystrophy, has been plagued by problems with side effects and adverse…
According to a story from The Jerusalem Post, Adrian Krainer is a prominent molecular geneticist and biochemist. His research was also pivotal in developing the first-ever disease modifying treatment for…
According to a publication from Fabry Disease News, the U.S. Food and Drug Administration (FDA) has approved American biotechnology company Avrobio's Investigational New Drug (IND) application for experimental Fabry disease…
Coronado is a resort city located in San Diego, California. It's known for its luxury hotels, delicious restaurants, beautiful parks, and gorgeous beaches. It has just been announced that the…
May is Cystic Fibrosis (CF) awareness month! CF is one of the more devastating rare diseases in the community, often affecting children and young people. So let's be extra mindful…
SCID Severe combined immunodeficiency (SCID) is a rare disease which causes patients to have little to no immune response. With practically no immune system, SCID patients are very susceptible to…
May is Healthy Vision Month! Rare disease or not, maintaining healthy eye health should be a priority for all of us. The National Institute of Health has resources for not…
The Origins of Fighting H.A.R.D. The Fighting H.A.R.D. Foundation was established in 2015 by two sisters who both live with multiple rare diagnoses. 16-year-old Allie is diagnosed with juvenile rheumatoid…
Google Backs Startup For Treating Heart Disease Via CRISPR Injections May 16, 2019 --Sharing is Caring-- Google Backs Startup For Treating Heart Disease Via CRISPR Injections Once in our life…
According to a story from BioSpace, the drug developer Biogen recently released interim results from a Phase 1/2 clinical trial. This clinical trial is testing the drug tofersen, which is…
This week, a press release from the office of South Carolina senator Lindsey Graham announced the designation of May 17th as Glioblastoma Awareness Day. The Senate Resolution was cosponsored by…
According to a story from Global Genes, the drug developer Viela Bio recently issued a presentation of the results from a clinical trial that tested the company's investigational product candidate…
PREFER PREFER stands for Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle. It is a multi-stage project developed by Newcastle University's Welcome Centre for Mitochondrial Research in effort…
According to a story from Pulmonary Hypertension News, a recent study found that patients with disease-associated pulmonary arterial hypertension had similar rates of survival compared to patients with idiopathic pulmonary…
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An estimated thirty million people in the U.S. are affected by chronic kidney disease. That translates into one out of three adults who are at risk. The majority, however,…
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