FDA Clears IND Application for Experimental CAR T-Cell Therapy in Myasthenia Gravis
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FDA Clears IND Application for Experimental CAR T-Cell Therapy in Myasthenia Gravis

The biotechnology company Cabaletta Bio has recently announced that the company investigational new drug (IND) application for its experimental CD19-CAR T-cell therapy CABA-201 has been cleared by the US Food…

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Experimental Drug Shows Promise in Prurigo Nodularis and Chronic Spontaneous Urticaria
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Experimental Drug Shows Promise in Prurigo Nodularis and Chronic Spontaneous Urticaria

According to a story from BioPharma Dive, the pharmaceutical company Celldex Therapeutics recently released positive results from two clinical trials. These trials involved the company's investigational therapy, barzolvolimab. In a…

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Study: The Effect of Patient Support Groups on Pulmonary Hypertension Patients and their Caregivers
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Study: The Effect of Patient Support Groups on Pulmonary Hypertension Patients and their Caregivers

Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…

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Study: Detection of Lysosomal Acid Lipase Deficiency in Japan FH Patients
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Study: Detection of Lysosomal Acid Lipase Deficiency in Japan FH Patients

In a study published in the scientific journal Atherosclerosis, a team of researchers sought to screen patients in Japan who had abnormally high LDL cholesterol (LDL-C) and a suspected diagnosis of familial…

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Phase III Trial meets Primary Endpoint for Patients with Eosinophilic Granulomatosis with Polyangiitis (EGPA)
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Phase III Trial meets Primary Endpoint for Patients with Eosinophilic Granulomatosis with Polyangiitis (EGPA)

  Patients with severe asthma with an eosinophilic phenotype who are 12 years of age or older may now receive treatment with FASENRA as an add-on maintenance treatment. Note that…

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Wealthy Facioscapulohumeral Muscular Dystrophy Patient Invests $100 Million to Find a Cure  

  Chip Wilson, billionaire founder of Lululemon Athletica, was first diagnosed with muscular dystrophy in 1987 at the age of 32. Wilson, who is now 66 years old told Global…

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Encouraging Findings in Phase 2 Gastric Cancer Clinical Trial
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Encouraging Findings in Phase 2 Gastric Cancer Clinical Trial

  Arcus Biosciences and Gilead Sciences issued a press release announcing encouraging overall response rates and six-month progression-free survival rate for their combination treatment of domvanalimab plus zimberelimab and chemotherapy…

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Scientists Demonstrate Gut Microbiome Connection with Transfer of Alzheimer’s to Healthy Animals
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Scientists Demonstrate Gut Microbiome Connection with Transfer of Alzheimer’s to Healthy Animals

  Scientists have previously suggested that the gut microbiome plays a role in causing Alzheimer’s disease, and this has now been confirmed. A recent report from ScienceAlert covered a study…

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Promising Interim Data in Phase 1/2 Hunter Syndrome Trial
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Promising Interim Data in Phase 1/2 Hunter Syndrome Trial

Professionals from all corners of the world convene at SSIEM each year to discuss the latest groundbreaking discoveries. According to a report in BioSpace, one such breakthrough was presented by…

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Scientists Are Calling Monkey’s Two-Year Survival After a Pig Kidney Transplant “Unprecedented”
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Scientists Are Calling Monkey’s Two-Year Survival After a Pig Kidney Transplant “Unprecedented”

  The xenotransplant involved transplanting Yucatan miniature pig kidneys into macaque monkeys with 69 genetic modifications. One monkey survived for over two years, offering hope to the team of Harvard…

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Study: Pelvic Floor Muscle Training is no Better Than Bladder Training for Overactive Bladder
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Study: Pelvic Floor Muscle Training is no Better Than Bladder Training for Overactive Bladder

According to a story from Medical Xpress, a recent study found that pelvic floor muscle training provided no additional advantage over bladder training in women with symptoms of overactive bladder.…

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Over 200 Rare Diseases to be Included in Genomics England Research Study
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Over 200 Rare Diseases to be Included in Genomics England Research Study

Genomics is revolutionizing healthcare processes, offering the potential to enhance the lives of numerous individuals by enabling the early detection of treatable disorders and providing lifesaving therapies. Every year, thousands…

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ALD and MLD to be Screened in Newborns in UK Generation Study
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ALD and MLD to be Screened in Newborns in UK Generation Study

Patient Worthy partner Alex TLC has announced that, in a large-scale newborn screening study supported by the UK's NHS and Genomics England, adrenoleukodystrophy (ALD) and metachromatic leukodystrophy (MLD) will be…

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