Patients Recruited for Phase 2 Study of Experimental Friedreich’s Ataxia Treatment

According to a press release from Barcelona-based biotechnology company Minoryx Therapeutics, the Company has completed enrollment for a phase 2 clinical study of its experimental Friedreich's ataxia treatment, MIN-102 (generic…

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Progressive Fibrosing Interstitial Lung Disease Drug Candidate Ofev Granted Breakthrough Therapy Status
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Progressive Fibrosing Interstitial Lung Disease Drug Candidate Ofev Granted Breakthrough Therapy Status

According to a publication from Scleroderma News, the American Food and Drug Administration (FDA) recently granted Breakthrough Therapy status to Ofev (generic name nintedanib), as a treatment for progressive fibrosing…

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Phase 1 Study of Experimental Systemic Sclerosis Treatment Bermekimab Begins
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Phase 1 Study of Experimental Systemic Sclerosis Treatment Bermekimab Begins

According to a recent press release from XBiotech, a Texas-based biotechnology company, patient dosing is now underway in a phase 1 clinical study of the Company's investigational systemic sclerosis treatment, bermekimab.…

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Experimental Facioscapulohumeral Dystrophy Drug Found Safe and Tolerable in Phase 1 Study
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Experimental Facioscapulohumeral Dystrophy Drug Found Safe and Tolerable in Phase 1 Study

A recent press release from Fulcrum Therapeutics, a Massachusetts-based biotechnology company, announced preliminary findings of the company's phase 1 clinical study of losmapimod in the treatment of facioscapulohumeral dystrophy. Losmapimod is…

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First Patient Dosed in Phase I/II Study of Experimental Gene Therapy for Cystinosis
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First Patient Dosed in Phase I/II Study of Experimental Gene Therapy for Cystinosis

According to a recent press release from the Massachusetts-based biotechnology company Avrobio, Inc., the first patient has been dosed in the Company's phase I/II clinical study of their investigational cystinosis…

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Last Patient Dosed in Phase 2 Study of Experimental ALS Treatment

In a recent press release, Massachusetts-based biotech company Amylyx Pharmaceuticals announced the completion of dosing in the 24 week study of their experimental amyotrophic lateral sclerosis treatment AMX0035. About Amyotrophic…

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FDA Approves Supplementary Treatment For Parkinson’s Disease “Off” Episodes
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FDA Approves Supplementary Treatment For Parkinson’s Disease “Off” Episodes

A recent publication from the US Food and Drug Administration announced the regulatory authority's approval of Nourianz (generic name istradefylline) for Parkinson's patients during "off" episodes. "Off" episodes are windows…

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Study Links Age of Onset and Year of Birth to Delayed Diagnosis for Hereditary Angioedema
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Study Links Age of Onset and Year of Birth to Delayed Diagnosis for Hereditary Angioedema

According to a study recently published in Allergy and Asthma Proceedings, certain risk factors may increase an individual's likelihood of receiving a late diagnosis for hereditary angioedema. Hereditary angioedema is…

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Positive Findings in Phase 2 Study of Refractory Status Epilepticus Drug Candidate
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Positive Findings in Phase 2 Study of Refractory Status Epilepticus Drug Candidate

According to a recent press release from the Pennsylvania-based Marinus Pharmaceuticals, the Company is reporting positive results of its phase 2 study of "ganaxolone," an investigational GABAA modulator for patients…

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More than 4% of People May be Immunocompromised – How Should They Approach Flu Season?
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More than 4% of People May be Immunocompromised – How Should They Approach Flu Season?

A recent publication from Healio detailed some of the challenges immunocompromised individuals might face during flu season. Influenza is a common and sometimes serious public health concern around the world, and…

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Researchers Identify Link Between Immune Cells and Genetic Signatures Associated with Autoimmune Disease
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Researchers Identify Link Between Immune Cells and Genetic Signatures Associated with Autoimmune Disease

According to a publication from EurekAlert, a study recently published in the medical journal Nature Genetics suggests how certain immune cells express genetic activity similar to that of variations of the…

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According to New Study, Signs and Symptoms of Kawasaki Disease May Differ in Infants
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According to New Study, Signs and Symptoms of Kawasaki Disease May Differ in Infants

A recent publication from BMC Pediatrics outlines a case study of Kawasaki disease in a group of infants from Italy. Because Kawasaki disease is a rare disease (with only an…

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ICYMI: FDA Approves Expanded Access Application of Experimental Niemann-Pick Type C Drug
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ICYMI: FDA Approves Expanded Access Application of Experimental Niemann-Pick Type C Drug

According to a press release from CTD Holdings, the American Food and Drug Administration (FDA) recently approved an Expanded Access application from a physician to treat a pediatric Niemann-Pick disease…

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Eosinophilic Asthma Treatment Nucala has FDA Approval Expanded to Children Aged 6 to 11
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Eosinophilic Asthma Treatment Nucala has FDA Approval Expanded to Children Aged 6 to 11

According to a press release from global pharmaceuticals titan GlaxoSmithKline, the US Food and Drug Administration (FDA) recently expanded approval of the Company's eosinophilic asthma treatment Nucala (generic name mepolizumab)…

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Specialty Pharmacists Play Important Role in Treatment of Primary Immunodeficiency Patients
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Specialty Pharmacists Play Important Role in Treatment of Primary Immunodeficiency Patients

A recent publication from Specialty Pharmacy Times noted that the rapidly-growing number of available human immunoglobulin treatments for primary immunodeficiency disorders could pose challenges for specialty pharmacists when treating patients.…

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Michigan Researchers to Study Neurofilament Light Chain Blood Levels as a Predictor of Multiple Sclerosis
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Michigan Researchers to Study Neurofilament Light Chain Blood Levels as a Predictor of Multiple Sclerosis

According to a publication from BioSpace, researchers from Michigan's Memorial Healthcare Institute for Neurosciences and Multiple Sclerosis are taking full advantage of recent technological advances in neurofilament light chain testing…

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NHS Finally Reverses Almost 2 Year Policy Against Offering Cerliponase Alfa to Batten Disease Patients
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NHS Finally Reverses Almost 2 Year Policy Against Offering Cerliponase Alfa to Batten Disease Patients

According to a recent publication from Express Digest, England's National Health Service (NHS) has reversed its earlier position on a rare disease drug after lengthy public debate, now agreeing to…

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FDA Begins Review of Biologics License Application for Neuromyelitis Optica Spectrum Disorder Drug
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FDA Begins Review of Biologics License Application for Neuromyelitis Optica Spectrum Disorder Drug

According to a press release from Maryland-based Viela Bio, the US Food and Drug Administration (FDA) recently agreed to review the Company's Biologics License Application for its neuromyelitis optica spectrum…

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Biomedical Innovation Law Expert Presents Ethical Difficulties Posed by Gene Editing

According to a publication from Charcot Marie Tooth News, biomedical law expert Timo Minssen recently gave a presentation at the New York Genome Center where he discussed the potential ethical…

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Zogenix Acquires Modis in $400 Million Buyout, Adds Experimental TK2 mDNA Depletion Syndrome Treatment to Pipeline

According to a press release from California-based biotechnology company Zogenix, the Company recently completed a multi-million dollar acquisition of Modis Therapeutics. Before its acquisition, Modis focused on the development of…

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Patient Advocacy Groups Call for Indian Health Ministry to Implement Interim Care Policy for Rare Disease Patients
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Patient Advocacy Groups Call for Indian Health Ministry to Implement Interim Care Policy for Rare Disease Patients

According to a recent publication from Moneycontrol, patient advocacy groups in India are calling for the country's Minister of Health and Family Welfare, Harsh Vardhan, to establish a provisional system…

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Korean Company Predicts Your Risk of Developing Certain Genetic Conditions by Sequencing Your Genome
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Korean Company Predicts Your Risk of Developing Certain Genetic Conditions by Sequencing Your Genome

According to a publication from the Korea Times, the Eone Diagnomics Genome Center in Incheon, South Korea, is working to improve patients' accessibility to their own genetic sequence. Researchers believe…

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