New Discovery Adds Greater Complexity to the Genetic Basis of Mitochondrial Disease
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New Discovery Adds Greater Complexity to the Genetic Basis of Mitochondrial Disease

According to a story from news-medical.net, a recent study has reshaped the scientific understanding of the origins of mitochondrial disease. For decades any mutation that affected the mitochondrial DNA and…

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Biotech Companies are Increasingly Focused on Deadly Diseases With Significant Unmet Need

According to a story from finanznachrichten.de, biotechnology companies are starting to set their sights on treating deadly diseases that are lacking effective treatment options, including some diseases that are considered…

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Preliminary Data from Phase 1 Trial for Multiple Myeloma Indicates Complete Response in a Patient

Phase 1 Trial A Phase 1 trial by Gamida Cell is examining a new potential treatment option for patients with multiple myeloma and relapsed/refractory non-Hodgkin's lymphoma who have not responded…

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First Patient Enrolled in Phase 2 Trial for Experimental RRMS Drug
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First Patient Enrolled in Phase 2 Trial for Experimental RRMS Drug

According to a publication from Multiple Sclerosis News Today, German biotechnology company Immunic Therapeutics has successfully enrolled the first participant for its phase 2 clinical trial for experimental relapsing-remitting multiple…

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Proof-of-Concept Trial for X-Linked Chronic Granulomatous Disease Treatment Shows Promise

According to a story from Markets Insider, the biopharmaceutical company Orchard Therapeutics recently announced the presentation of proof-of-concept data in regards to their investigational drug candidate OTL-102. This experimental therapy…

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Observational Study Conducts Long Term Follow up of Niemann-Pick Disease Type B Patients
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Observational Study Conducts Long Term Follow up of Niemann-Pick Disease Type B Patients

A study published in the Orphanet Journal of Rare Diseases monitored the long-term progression of 16 patients that had Niemann-Pick Disease type B. The study served to gather important observational data…

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International Genetic Research Group to Begin Study of Hypermobile Ehlers-Danlos Syndrome (hEDS)

A recent announcement by the Ehlers-Danlos Society marks the beginning of the recruitment phase for an international study devoted to determining the root cause of hEDS. The gathering of this…

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Bioscience Companies in Ohio Take Aim at Rare Diseases
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Bioscience Companies in Ohio Take Aim at Rare Diseases

According to a story from BioPortfolio, Rare Disease Day, which took place on February 28th, 2019, serves as a time of reflection on the progress that has been made in…

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A Beta Thalassemia Patient will be the First Dosed with CRISPR-Based Gene Therapy in Company Sponsored Trial
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A Beta Thalassemia Patient will be the First Dosed with CRISPR-Based Gene Therapy in Company Sponsored Trial

According to a story from MedCity News, a patient with the rare blood disorder beta thalassemia will be the first patient dosed in a Phase I/II clinical trial involving a…

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Experimental Treatment for Bronchopulmonary Dysplasia Shows Long Term Positive Impact in Rat Model
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Experimental Treatment for Bronchopulmonary Dysplasia Shows Long Term Positive Impact in Rat Model

According to a story from globenewswire.com, the drug developer The Cell Factory and the University of Padova in Italy have been conducting research in order to demonstrate the effectiveness of…

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Experimental Treatment Could be Potential Breakthrough for Generalized Pustular Psoriasis
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Experimental Treatment Could be Potential Breakthrough for Generalized Pustular Psoriasis

According to a story from dubainewstoday.blogspot.com, the data from a Phase I clinical trial suggests that an experimental drug candidate from the pharmaceutical company Boehringer Ingelheim could substantially improve the…

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MD Anderson Physicians Use a Diagnostic Method Called Sentinel Lymph Node Biopsy To Determine Spread of Cancer
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MD Anderson Physicians Use a Diagnostic Method Called Sentinel Lymph Node Biopsy To Determine Spread of Cancer

Although some ultrasound and/or CT scan studies may show that patients’ lymph nodes (glands) are free of disease, upon further examination using sentinel lymph node biopsies,(SLNB) the doctors at MD…

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Study Investigates the Most Predictive Indicators of Bronchopulmonary Dysplasia

What is Bronchopulmonary Dysplasia? Bronchopulmonary Dysplasia (BPD) is a diagnosis most commonly given to premature newborns. In children, BPD is also the most common cause of Pulmonary Hypertension (PH). Premature babies often develop…

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