According to a story from koco.com, the community of Ada, Oklahoma, recently held a birthday celebration for a local affected by Alexander disease. Hunter Goodwin recently turned 18 years old,…
According to a story from Biospace, the RNAi therapeutics company Alnylam Pharmaceuticals Inc. recently announced that its experimental product candidate vutrisiran has earned Fast Track designation from the US Food…
According to a story from PR Newswire, the National Organization for Rare Disorders (NORD) has just recently kicked off a COVID-19 Critical Relief Program. In alignment with its mission to…
The ongoing coronavirus/COVID-19 pandemic has caused widespread uncertainty across the globe, and a lot of us have questions about what to do or what to expect. The rare disease community…
Según una historia de CureDuchenne, la compañía de biotecnología Dyne Therapeutics anunció recientemente que CureDuchenne Ventures ha realizado una inversión de capital que respaldará el desarrollo de todas las nuevas…
April is Sjögren's Syndrome Awareness Month. This is held annually in an effort to spread awareness about this syndrome, and has been held since 1998. Unfortunately, this disease receives little…
According to a story from Porphyria News, Lucia Monaco, PhD, is the chair of the International Rare Diseases Research Consortium (IRDiRC). Despite the ongoing disruptions caused by the coronavirus/COVID-19 pandemic,…
According to a story from Spectrum News, the Andrews family has been dealing with a rare disease called Niemann-Pick disease type C for years. Parents Pam and Chris knew early…
De acuerdo a una historia de Fierce Biotech, recientemente ha habido algunos avances impresionantes en el tratamiento de la fibrosis quística. El último hito fue desarrollar y aprobar el medicamento…
In a story written for The Washington Post, Jessica Slice writes about a frightful predicament for patients with chronic illnesses that cause disability. Jessica lives with dysautonomia and hypermobile Ehlers-Danlos…
A press release from the US Food and Drug Administration (FDA) recently announced the approval of the drug selumetinib (marketed as Koselugo) for the treatment of patients aged two years…
According to a story from XTalks, pulling off a clinical trial testing a new drug in a scientifically rigorous manner is no simple feat. It requires the triangulation of a…
According to a study published in BMC Pediatrics, the rare disease known as Felty's syndrome is primarily known to affects adults between the ages of 50 and 70, but on very rare…
According to a story from globenewswire.com, the biopharmaceutical company Liquidia Technologies, Inc. just announced that the company's New Drug Application (NDA) has been officially accepted by the US Food and…
According to a story from Malvern Daily Record, the biopharmaceutical company Millendo Therapeutics, Inc. recently released data from its phase 2b clinical trial. This study was testing the company's experimental…
As Patient Worthy readers, we hope you and your families are doing well during this challenging time. The health and safety of our rare community is always at the…
According to a story from People's Pharmacy, a dizzying array of advice related to minimizing spread or coronavirus/COVID-19 has barraged our senses over the last few weeks as the number…
According to a study published in the journal Neurology, a measure called the vastus lateralis fat fraction was found to be useful in predicting the age at which a patient living…
According to a story from gurufocus.com, the biotechnology company Capricor Therapeutics has recently announced the initiation of a compassionate use program for its experimental drug CAP-1002. This drug will be…
According to a study published in January in the scientific journal Acta Neurologica Scandinavica, the use of gamma knife surgery as a treatment for a rare tumor called a vestibular schwannoma can…
April is designated as Sarcoidosis Awareness Month. This year's event will be the sixth annual awareness campaign, which will involve collaboration between the Foundation for Sarcoidosis Research and the American…
According to a story from BioSpace, Acceleron Pharma Inc. and Bristol Myers Squibb have announced recently that the US Food and Drug Administration (FDA) has recently approved a new drug…
April 17th, 2020 is World Hemophilia Day. This event is held to help foster awareness about hemophilia and other related bleeding disorders. It is a day when this rare community…
The month of April is recognized as Parkinson's Disease Awareness Month. While Parkinson's disease is not considered rare, Patient Worthy chooses to continue to provide coverage of this illness because…
April 22nd, 2020 heralds the beginning of World Primary Immunodeficiency (PI) Week. This will be the ten year anniversary of the event, which is designed to spread awareness about PI…
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