Drug Shows Potential in Treating Hyperkalemia Associated With End-Stage Kidney Disease
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Drug Shows Potential in Treating Hyperkalemia Associated With End-Stage Kidney Disease

According to a story from BioPortfolio, the biopharmaceutical company AstraZeneca recently announced the release of positive results from a phase 3b clinical trial. This trial was testing the safety and…

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Study Finds Poor Adherence to PDE-5I Drugs Can Have Consequences in Pulmonary Arterial Hypertension
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Study Finds Poor Adherence to PDE-5I Drugs Can Have Consequences in Pulmonary Arterial Hypertension

According to a story from Pulmonary Hypertension News, a recent study has found that lapses in treatment adherence can result in serious side effects for pulmonary arterial hypertension (PAH) patients…

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Chronic Lymphocytic Leukemia: Patterns of Treatment, Costs, and Adverse Events
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Chronic Lymphocytic Leukemia: Patterns of Treatment, Costs, and Adverse Events

According to a story from Cancer Therapy Advisor, a recent study published in the journal Cancer Medicine indicated that chronic lymphocytic leukemia (CLL) patients who dealt with adverse events during treatment…

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Patient Recruitment Completed for Phase 2 Study of Mucopolysaccharidosis VI Drug Candidate

According to a press release from the French biotechnology company Inventiva, the company has completed recruitment for a phase 2 clinical trial evaluating its experimental mucopolysaccharidosis VI drug, odiparcil. About…

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Study Identifies Differences in Progression and Onset Between Type 4B1 and 4B2 Charcot-Marie-Tooth Disease
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Study Identifies Differences in Progression and Onset Between Type 4B1 and 4B2 Charcot-Marie-Tooth Disease

According to a story from Charcot-Marie-Tooth News, a recent study was conducted that compared the presentation of symptoms between two different variants of Charcot-Marie-Tooth disease (CMT): CMT4B1 and CMT4B2. The…

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A New Way to Study Disease: Scientists Have Recreated a Blood-Brain Barrier Outside the Body
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A New Way to Study Disease: Scientists Have Recreated a Blood-Brain Barrier Outside the Body

  The blood-brain barrier is a “gatekeeper” that prevents toxins and foreign matter in the bloodstream from traveling to the brain. Check Orphan recently carried an article that had been…

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Tips by a Myelodysplastic Syndromes Patient on How to Navigate the Internet When Researching Your Rare Disease

Jane Biehl Jane Biehl has worked as a professor, rehabilitation counselor, and a librarian. She holds three different degrees, and spreads her knowledge through freelance writing. She also has a…

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Study Confirms the Superiority of Rituximab (Rituxan) in Treating Pemphigus Vulgaris
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Study Confirms the Superiority of Rituximab (Rituxan) in Treating Pemphigus Vulgaris

According to a story from marketscreener.com, the biotechnology company Roche recently announced the results from a company sponsored phase 3 clinical trial that compared the safety and efficacy of rituximab…

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The New 5F9 Antibody Looks Encouraging as it Targets MDS and AML Leukemia in a Phase 1b Clinical Trial

  The California Institute for Regenerative Medicine (CIRM) has funded a study by a company called Forty Seven, Inc. The clinical trial is investigating the benefits of a drug therapy for two cancers…

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New Partnership Aims to Develop New Mobility Controls for Amyotrophic Lateral Sclerosis Patients
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New Partnership Aims to Develop New Mobility Controls for Amyotrophic Lateral Sclerosis Patients

According to a story from ucf.edu, a new partnership between Mayo Clinic and Limbitless Solutions is planning initiate a new clinical trial that aims to improve mobility options for patients…

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Researchers Identify New Mutations Linked to Charcot-Marie-Tooth Disease
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Researchers Identify New Mutations Linked to Charcot-Marie-Tooth Disease

According to a story from Charcot-Marie-Tooth News, a team of scientists have successfully identified four new mutations that are capable of causing Charcot-Marie-Tooth disease. These mutations affect the GJB1 gene. These new…

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Experimental Treatment for Hunter Syndrome Earns Rare Pediatric Disease Designation and Orphan Drug Designation
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Experimental Treatment for Hunter Syndrome Earns Rare Pediatric Disease Designation and Orphan Drug Designation

According to a story from globenewswire.com, the biopharmaceutical company Denali Therapeutics, Inc. has recently announced that its experimental product candidate DNL310, which is currently being developed as a treatment for…

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Critical Research Into Amyotrophic Lateral Sclerosis Therapies Gets Funding Boost
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Critical Research Into Amyotrophic Lateral Sclerosis Therapies Gets Funding Boost

According to a story from Alabama News Center, a scientist affiliated with Southern Research, Dr. Rita Cowell, has been hard at work researching new potential treatments for amyotrophic lateral sclerosis,…

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Presentations at the ATS Conference Demonstrate the Value of the Pulmonary Fibrosis Foundation’s Patient Registry

At the 2019 American Thoracic Society Conference this year multiple presentations demonstrated the importance of the Pulmonary Fibrosis Foundation's Patient Registry. About the Registry This registry was created by the Pulmonary…

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