New Research Suggests “Nonsense” RNA Segment Might Play Important Role in Ankylosing Spondylitis
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New Research Suggests “Nonsense” RNA Segment Might Play Important Role in Ankylosing Spondylitis

According to a publication from Ankylosing Spondylitis News, a recent study published in BMC Musculoskeletal Disorders suggests that LINC00311, a lengthy RNA molecule, is over-expressed in individuals with ankylosing spondylitis.…

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Researchers Ask Hemophilia Patients to Evaluate Their Quality of Life on Study Questionnaire
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Researchers Ask Hemophilia Patients to Evaluate Their Quality of Life on Study Questionnaire

According to a publication from Hemophilia News Today, an international team of researchers with members from France and the United Kingdom recently conducted a study of over 100 hemophilia patients…

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Recent Study Finds Better Than Expected Safety Data for TNF Inhibitors in Ankylosing Spondylitis
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Recent Study Finds Better Than Expected Safety Data for TNF Inhibitors in Ankylosing Spondylitis

According to a story from Ankylosing Spondylitis News, a recent study has found that the class of drugs known as tumor necrosis factor (TNF) inhibitors may actually be safer for…

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Minovia Kicks Off Phase I/II Trial of Experimental Pearson Syndrome Treatment
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Minovia Kicks Off Phase I/II Trial of Experimental Pearson Syndrome Treatment

According to a publication from Business Wire, the Israeli biotechnology company Minovia Therapeutics recently finished dosing the first participant in a phase I/II clinical study of the Company's experimental mitochondrial…

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Study Identifies Broader Range of Clinical Presentation for Mitochondrial Disease
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Study Identifies Broader Range of Clinical Presentation for Mitochondrial Disease

According to a story from Wellcome Center Mitochondrial Research, a multi-center collaborative study that involved researchers from different sites around the UK has resulted in an improved understanding of the…

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Recent Study Indicates Lyso-Gb1 is an Extremely Effective Biomarker for Monitoring Children with Gaucher Disease

Gaucher disease (GD) is a rare lysosomal storage disorder. It is caused by a deficiency in the glucocerebrosidase enzyme. In June, a new study was published in the International Journal of…

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Biotech Companies Team Up to Launch MapKure, a New Joint Venture to Develop Small Molecule Anticancer Drug
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Biotech Companies Team Up to Launch MapKure, a New Joint Venture to Develop Small Molecule Anticancer Drug

According to a press release from BeiGene, the Company and SpringWorks Therapeutics have launched MapKure, LLC — a new, jointly-operated venture owned by both partners. MapKure was founded to develop…

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New Clinical Trial Application for Alpha-1 Antitrypsin Deficiency-Associated Liver Disease Submitted in Sweden

Dicerna Pharmaceuticals has just announced that they have submitted a Clinical Trial Authorization application for their investigational therapy DCR-A1AT. This application has been submitted to the Swedish Medical Products Agency…

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Company Starts up Late Stage Trial Testing Experimental WHIM Syndrome Treatment
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Company Starts up Late Stage Trial Testing Experimental WHIM Syndrome Treatment

According to a story from BioPortfolio, the biopharmaceutical company X4 Pharmaceuticals, Inc. has recently initiated a phase 3 clinical trial that is testing its current lead product candidate mavorixafor. This…

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Early Data Released From Phase 1B Duchenne Muscular Dystrophy Gene Therapy Trial
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Early Data Released From Phase 1B Duchenne Muscular Dystrophy Gene Therapy Trial

According to a story from BioPortfolio, the drug company Pfizer has recently presented data from its phase 1b clinical trial that tested the company's experimental gene therapy PF-06939926, which is…

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Researchers Observe First Cases of Congenital Leptin Deficiency in the Americas
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Researchers Observe First Cases of Congenital Leptin Deficiency in the Americas

According to a press release from the Texas Biomedical Research Institute (Texas Biomed), researchers from the Institute have identified another new genetic mutation that they believe can cause rare congenital…

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Canadian Study Indicates That Prescribing Practices of Infliximab for Ulcerative Colitis Need Amendment

Inflammatory Bowel Diseases Inflammatory bowel diseases (IBD) is the overarching term for both ulcerative colitis and Crohn's disease. In Canada, there are around 270,000 individuals living with the one of…

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Scientists Identify Possible Link Between Pancreatic Cancer and Muscle Wasting Condition Cachexia
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Scientists Identify Possible Link Between Pancreatic Cancer and Muscle Wasting Condition Cachexia

According to a publication from EurekAlert, researchers from the University of Oklahoma College of Medicine (OUCM) recently published a study in the journal Gastroenterology suggesting how cachexia is triggered in…

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Pancreatic Cancer Medication Shows Strong Promise Treating Advanced Biliary Tract Cancers
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Pancreatic Cancer Medication Shows Strong Promise Treating Advanced Biliary Tract Cancers

According to a publication from the Arizona BioIndustry Association, Dr. Rachna Shroff and a team of researchers from the Arizona Cancer Center are studying the use of nab-paclitaxel (a pancreatic…

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King’s College Research Suggests Serotonin Levels Could Predict Parkinson’s Years Before Symptoms
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King’s College Research Suggests Serotonin Levels Could Predict Parkinson’s Years Before Symptoms

According to a publication from the BBC, researchers with King's College London believe they've identified early biomarkers for Parkinson's disease that could help physicians predict who will be affected up…

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CIRM Awards $12 Million Grant for Clinical Study of Gene Therapy for Cystinosis Patients
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CIRM Awards $12 Million Grant for Clinical Study of Gene Therapy for Cystinosis Patients

According to a press release from the California Institute for Regenerative Medicine (CIRM), the Institute's governing body has approved a grant of nearly $12 million to University of California, San…

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