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Rare Classroom: Glioblastoma Multiforme

Rare Classroom: Glioblastoma Multiforme

Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…

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Modified Base Editing Could Correct DMD Mutations

Modified Base Editing Could Correct DMD Mutations

Genome editing is a topic which has been on the forefront of medical discussion for years. After all, gene editing allows for the potential to treat - or even cure…

Continue Reading Modified Base Editing Could Correct DMD Mutations
Here’s Why You Should Be Aware of Rhabdo This Summer

Here’s Why You Should Be Aware of Rhabdo This Summer

People are rushing to get in shape for summer; it's common to see the gyms a little more full than normal. While exercise is good for you, it's important not…

Continue Reading Here’s Why You Should Be Aware of Rhabdo This Summer
Phase 3 International Trial for Transthyretin Amyloid Cardiomyopathy is Fully Enrolled

Phase 3 International Trial for Transthyretin Amyloid Cardiomyopathy is Fully Enrolled

Researchers have just announced that they have completed enrollment for a new Phase 3 trial for patients with transthyretin-mediated (ATTR) amyloidosis who have heart disease. This trial is examining a…

Continue Reading Phase 3 International Trial for Transthyretin Amyloid Cardiomyopathy is Fully Enrolled
Investigative Therapy for Endogenous Cushing’s Syndrome is Being Evaluated by The FDA

Investigative Therapy for Endogenous Cushing’s Syndrome is Being Evaluated by The FDA

Strongbridge Biopharma has just announced that the FDA has decided to review the New Drug Application for RECORLEV, a therapy for endogenous Cushing's syndrome. The target action date is January…

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Remarkable Results From a Phase 2 Trial for Beta Thalassemia

Remarkable Results From a Phase 2 Trial for Beta Thalassemia

As reported in Biospace; a Phase 2 study by Bristol-Myers Squibb and Acceleron Pharma Inc. earned itself recognition as among the top six abstracts received by the Presidential Symposium of…

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Compassion Corner: Compassion, Empathy, Sympathy, and Control in a Clinical Setting

Compassion Corner: Compassion, Empathy, Sympathy, and Control in a Clinical Setting

Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…

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Using High-Tech Nanoparticles to Treat ACD Pulmonary Issues

Using High-Tech Nanoparticles to Treat ACD Pulmonary Issues

Alveolar capillary dysplasia with misalignment of the pulmonary veins (ACD/MPV), or alveolar capillary dysplasia (ACD) for short, is a rare, congenital, and life-threatening disorder. Without receiving lung transplants in infancy,…

Continue Reading Using High-Tech Nanoparticles to Treat ACD Pulmonary Issues
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Finding Light Through Story-The Power of Ambassadorship in the Endometrial Cancer Community
*cancer that cannot be removed with surgery or has spread to other parts of the body. GEJ= gastroesophageal junction (where the esophagus joins the stomach)
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