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An article appeared recently in BioNews announcing the first-ever approval of an RNA-based therapy for use on the British National Health Service. The name of the drug is Patisiran…
According to a story from finanznachricten.de, the drug company Novartis recently announced that the European Medicine Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive…
According to a story from BioSpace, the biopharmaceutical company BeiGene Co., Ltd has recently announced that its drug zanubrutinib (marketed as BRUKINSA) has earned Accelerated Approval from the US Food…
For people with sickle cell disease, a common, painful symptom is vaso-occlusive crisis, which is when blood circulation is blocked by sickled red blood cells. This restriction of circulation results…
Researchers at the University of Pennsylvania are working on a new potential gene therapy for Duchenne muscular dystrophy (DMD). DMD is a rare form of muscular dystrophy that primarily affects…
By Rachel Whetstone from In the Cloud Copy Although rare diseases are uncommon by definition, a new study shows that the total number of people affected by rare diseases is…
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“Myasthenia” means “muscular weakness”. According to a recent article in The Star, studies in Asia indicate that there are approximately five new cases of myasthenia gravis (MG) reported each…
According to a story from pm360online.com, the biopharmaceutical company miRagen Therapeutics, Inc., recently presented Phase I clinical trial data regarding the company's experimental drug cobomarsen. This data was presented at…
According to a story from Pulmonary Advisor, the rare condition pulmonary arterial hypertension (PAH) is mostly known for the impacts that it can have on the lungs and on the…
According to a story from BioPortfolio, the biopharmaceutical company GW Pharmaceuticals plc recently announced that its medicine Epidyolex has earned recommendation from the UK National Institute for Health and Care…
According to a story from metro.co.uk, Harley Bond was three years old when he was first diagnosed with Sanfilippo syndrome type B, a rare progressive genetic disorder. Now five years…
According to a story from the Malvern Daily Record, the biotechnology company Exelixis, Inc. has recently announced that its partner company Ipsen BioPharmaceuticals Canada Inc. has had its drug cabozantinib…
This year, Patient Worthy will have the privilege to cover one of the planet's foremost patient centered events on chronic pain: The International Pain Summit, which will be held from…
In an article from the Orphanet Journal of Rare Diseases, a recent study provides an overview of the treatment of familial Mediterranean fever (FMF) that does not respond to the standard…
According to a story from the US Food and Drug Administration (FDA), Dr. Amy Abernethy, who is the current Prinicipal Deputy Commissioner at the agency, was eager to start working…
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An article by Dr. Syavra Tipirneni, dermatologist, recently appeared in BioSpectrum News. Dr. Tipirneni points out that we often succumb to our desire for perfect skin without the realization…
According to a story from PR Newswire, the clinical-stage company Vyriad, Inc. and the biotechnology company Regeneron Pharmaceuticals, Inc. are partnering in a licensing agreement and research collaboration to investigate…
According to a story from WBUR, a preliminary evaluation of a young patients diagnosed with Tay-Sachs disease appears to show some encouraging signs. Two patients were treated with an experimental…
According to a story from Medscape, the drug burosumab (marketed as Crysvita) demonstrated its ability to provide long term benefits for nearly two years to patients with the rare disease…
According to a story from Targeted Oncology, the US Food and Drug Administration (FDA) has granted Fast Track designation to the experimental drug OMP-305B83, also known as navicixiumab. This designation…
According to a story from abc.net.au, parents Kellee and Jamie Clarkson of Queensland, Australia are calling for the implementation of newborn screening in the state for the rare disorder spinal…
Doctor Leah Kaminsky discusses a new era in healthcare in her recent article that offers hope through new technology. How Can AI Help? AI will be able to predict changes to…
In a story for Hemophilia News Today, author Shellye Horowitz has written a piece titled "An Open Letter to Providers Treating Women with Hemophilia." Women still face discrimination and unequal…
According to a story from Nature, Dr. Carlos Heras-Palou was diagnosed with hATTR amyloidosis when he was just 39 years old. Initially, the devastating diagnosis was completely overwhelming, as the disease…
According to a story from Daily Nation, Beneddettah Wangui of Kenya had been experiencing chronic pain since her teen years when she was finally diagnosed with fibromyalgia in 2012. However,…
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