Year Two of This Alport Syndrome Trial Concludes with Encouraging Findings
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Year Two of This Alport Syndrome Trial Concludes with Encouraging Findings

According to a story from GlobeNewswire, the biopharmaceutical company Reata Pharmaceuticals, Inc., recently announced that year two of its phase 3 clinical trial has been completed. This trial is testing…

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Study Identifies Clinical Characteristics of Chronic Nonbacterial Osteomyelitis
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Study Identifies Clinical Characteristics of Chronic Nonbacterial Osteomyelitis

According to a story from EurekAlert! a recent study presented at the annual meeting of the American College of Rheumatology describes some of the key clinical feature of chronic nonbacterial…

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Hereditary Angioedema Patients in the UK Get Early Access to New Treatment
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Hereditary Angioedema Patients in the UK Get Early Access to New Treatment

According to a story from GlobeNewswire, the company BioCryst Pharmaceuticals, Inc., recently announced that its drug berotralstat (marketed as Orladeyo) will be available in the UK under the Early Access…

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New Study Pioneers Method to Measure Myalgic Encephalomyelitis Severity
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New Study Pioneers Method to Measure Myalgic Encephalomyelitis Severity

A recent study published in the Journal of Translational Medicine describes an accurate and objective approach that aims to evaluate the severity of myalgic encephalomyelitis, also known as chronic fatigue syndrome. This…

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Experimental Treatment for Sickle Cell Disease Earns Orphan Drug Designation in the EU
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Experimental Treatment for Sickle Cell Disease Earns Orphan Drug Designation in the EU

According to a story from Street Insider, the biopharmaceutical company Forma Therapeutics Holdings, Inc., recently announced that its investigational therapy candidate FT-4202 has earned Orphan Drug designation from the European…

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Experimental Treatment for Acid Sphingomyelinase Deficiency Shows Potential in Trials
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Experimental Treatment for Acid Sphingomyelinase Deficiency Shows Potential in Trials

In a press release from Sanofi, a global biopharmaceutical company, the company's experimental therapy candidate olipudase alfa has demonstrated the ability to substantially improve spleen volume and lung function in…

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Study: Glomerular Diseases Linked to Greater Risk of Cardivascular Disease

According to a story from Newswise, a recent study concluded that adult patients living with glomerular diseases face an increased chance of cardiovascular disease as well. The findings were first…

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The Drug Dupixent Shows Potential as an Eosinophilic Esophagitis Treatment
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The Drug Dupixent Shows Potential as an Eosinophilic Esophagitis Treatment

According to a story from GlobeNewswire, the medication dupilumab (marketed as Dupixent®) has demonstrated potential efficacy in a phase 3 clinical trial as a treatment for eosinophilic esophagitis, a rare…

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Experimental Stargardt Disease Treatment Earns Orphan Drug and Rare Pediatric Disease Designations
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Experimental Stargardt Disease Treatment Earns Orphan Drug and Rare Pediatric Disease Designations

According to a story from PR Newswire, the biopharmaceutical company reVision Therapeutics, Inc. has just announced that its experimental therapy REV-0100 has received both Rare Pediatric Disease designation and Orphan…

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Experimental Drug Earns Orphan Drug Designation for Polymyositis and Dermatomyositis
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Experimental Drug Earns Orphan Drug Designation for Polymyositis and Dermatomyositis

According to a story from Biotech 365, the biotechology company Kezar Life Sciences, Inc. recently announced that its experimental therapy KZR-616 has earned Orphan Drug designations from the US Food…

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Case Report Describes First Known Ocular Myasthenia Gravis Case with COVID-19
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Case Report Describes First Known Ocular Myasthenia Gravis Case with COVID-19

According to a story from Myasthenia Gravis News, a recent report describes the first-ever documented case of a patient developing the ocular variant of myasthenia gravis in connection with COVID-19…

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This Organization is Offering Spinal Muscular Atrophy Patients Financial Aid for Treatment
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This Organization is Offering Spinal Muscular Atrophy Patients Financial Aid for Treatment

According to a story from SMA News Today, the Patient Access Network (PAN) Foundation announced that it is now offering financial assistance to help patients living with the rare disease…

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