Experimental Treatment Combination Shows Efficacy in Acute Myeloid Leukemia and Myelodysplastic Syndromes
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Experimental Treatment Combination Shows Efficacy in Acute Myeloid Leukemia and Myelodysplastic Syndromes

  Researchers acknowledge that the primary cause of failure of stem cell transplants is disease relapse. This holds true specifically for allogeneic hematopoietic stem cell transplants where a cancer patient…

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Diabetic Retinopathy: Tele-Ophthalmology and AI Equally Effective for Diagnosis
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Diabetic Retinopathy: Tele-Ophthalmology and AI Equally Effective for Diagnosis

According to a story from Ophthalmology Times, the COVID-19 pandemic rapidly expanded the usage of telemedicine, including virtual, tele-ophthalmology appointments. It also highlighted the effectiveness of AI, and both of…

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Nonprofit Drug Development is the Answer for Many Rare Disease Communities in Need of Treatment
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Nonprofit Drug Development is the Answer for Many Rare Disease Communities in Need of Treatment

Written By Ashely Winslow, PhD, President & Chief Scientific Officer of Odylia Therapeutics One of the biggest issues impacting many rare disease communities is the availability of approved treatments. While…

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ICYMI: Patients with a Certain Genotype Are More Likely to Discontinue Imuran Due to Toxicity

  Dr. Cecilia Chung and associates at the Vanderbilt University’s Medical Center conducted a study published recently in MedPage Today. Results showed that patients who had inflammatory disorders, as well…

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Results From This Study are Encouraging for Alpha-1 Antitrypsin Deficiency Liver Disease
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Results From This Study are Encouraging for Alpha-1 Antitrypsin Deficiency Liver Disease

  Takeda and its collaborator, Arrowhead Pharmaceuticals, recently announced results from AROAAT-2002 of the investigational drug fazirsiran. The drug is an RNA interference therapeutic developed to lessen alpha-1 antitirypsin protein…

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Azathioprine: Specific Genotype Linked to Treatment Discontinuation Due to Toxicity
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Azathioprine: Specific Genotype Linked to Treatment Discontinuation Due to Toxicity

According to a story from Medpage Today, patients using the drug azathioprine who carried a certain genotype were three times more likely to need to either cease treatment or reduce…

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Epilepsy Research UK Joins Tuberous Sclerosis Association in Funding Collaboration
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Epilepsy Research UK Joins Tuberous Sclerosis Association in Funding Collaboration

  According to a recent announcement, Epilepsy Research UK has announced a funding collaboration with the Tuberous Sclerosis Association which will support the investigation of tuberous sclerosis complex (TSC)-associated epilepsy.…

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Study Shows Keratoconus Patients Have Permanent Corneal Structure Changes If Unable to Get Timely Corneal Crosslinking
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Study Shows Keratoconus Patients Have Permanent Corneal Structure Changes If Unable to Get Timely Corneal Crosslinking

According to a recent article, a study has shown that keratoconus patients who do not get their timely corneal crosslinking will have permanent changes in their corneal structure.   Keratoconus…

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Charcot-Marie-Tooth Disease: Theophylline Boosts Myelin Production in CMT1A Mice
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Charcot-Marie-Tooth Disease: Theophylline Boosts Myelin Production in CMT1A Mice

According to a story from Charcot-Marie-Tooth News, a recent study using a mouse model of Charcot-Marie-Tooth disease type 1A produced some interesting results. The researchers found that administration of theophylline…

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ICYMI: Trabectedin for Ovarian Cancer Did Not Meet its Primary Endpoint
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ICYMI: Trabectedin for Ovarian Cancer Did Not Meet its Primary Endpoint

  Findings from the MITO23 Trial investigating the outcome of the chemotherapy drug trabectedin against physician’s choice to treat BRCA-mutated patients with ovarian cancer were presented at ASCO’s 2022 Annual…

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New Program Evaluates the Adaptability of Precision Medicine to Identify Over 400 Genetic Disorders and Potential Treatments
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New Program Evaluates the Adaptability of Precision Medicine to Identify Over 400 Genetic Disorders and Potential Treatments

  The novel program called BeginNGS™ was described in a recent article which has a goal of testing for five hundred disorders and several thousand infants. Rady Children's Institute for…

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Improved Genetic Testing Brings a More Accurate Diagnosis of Primary Immunodeficiency Disorders

  Genetic testing has always been costly and directed at DNA sequencing. Only a limited number of genes could be tested. It was useful to a small number of patients…

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