Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

    Personalized Medicine: A Drug Tailored for a Six Year Old With Batten Disease May Serve as a Template for Similar Rare Disorders

      Mila Makovec began her life as a normal, healthy infant, and according to a recent article in Science Magazine, she continued along this path until reaching the age of…

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    Found: Pelizaeus-Merzbacher Disease, the Cause, and a Potential Cure

      According to a recent article published by the University of California San Francisco, Pelizaeus-Merzbacher disease (PMD) is a rare neurological disease affecting young boys. PMD fatalities may occur before the child…

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    Studies Reveal How Blood Flow and Oxygen Delivery Are Affected by Sickle Cell Disease (SCD)

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    Physician and investigator John Wood, MD, Ph.D. of Childrens Hospital of Los Angeles specializes in studying the challenges facing SCD patients. He has currently focused his attention on how oxygen delivery…

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    Clinical Trials Show Promising Results for Risdiplam in Treatment of Spinal Muscular Atrophy Types 1, 2, and 3

    According to a recent article in SMA News Today, risdiplam (formerly RG7916)  is in ongoing studies of patients with spinal muscular atrophy (SMA). The drug is an investigational medicine developed by Genentech…

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    The Phase 3 Trial in Diabetic Peripheral Neuropathy May Have Failed Due to a Critical Mistake

      FierceBiotech recently reported that Helixmith Co., based in Korea and previously known as ViroMed, coordinated a Phase 3 clinical trial involving five hundred diabetic peripheral neuropathy (DPN) patients. The…

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    Step Therapy Supposedly Minimizes Risk, but for Brittany it Caused Severe Complications

      The Providence Journal recently published an article by Brittany Ricci, a student at Brown University’s Alpert Medical School, detailing her year-long struggle to receive the treatment her doctor had prescribed…

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    A New European Consortium Dedicated to Finding and Treating Rare Diseases

    A molecular biologist, Dr. Daria Julkowska, coordinates an international consortium in Paris that is largely funded through the 28-member European Union (EU). According to a report published in the Charcot-Marie-Tooth…

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    FDA Approval Was Granted For a Duchenne Muscular Dystrophy Drug Amid Internal Disagreements

      The accelerated approval granted by the FDA for the Duchenne muscular dystrophy drug eteplirsen, developed by Sarepta Therapeutics, was granted only after internal disagreements and protests had been resolved.…

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    FDA Approves a Combination of Drugs for Diffuse Large B-cell Lymphoma

      Diffuse large B-cell lymphoma  (DLBCL) is a common type of non-Hodgkins lymphoma. World Pharma News recently reported that the FDA approved the first chemoimmunotherapy, Polivy, for DLBCL in combination with…

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    The Sudden Closure of Northwestern University’s Stem Cell Therapy Clinic Raises Questions That Remain Unanswered

    According to a recent article in The Scientist, Northwestern Medicine’s successful autoimmune and immunotherapy program is winding down and will be closed by the end of the year. The program,…

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    Patients in Mayo Clinic’s RIGHT Study Carried One or More Genetic Variants That May Affect Their Response to Medication

      Pharmacogenomics plays an important role in predicting whether patients will benefit from certain medications or if the medication will cause serious side effects. The process involves the way in…

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    Biohacking: A Small Group of Amateur Biologists Are Hoping to Market Their Knockoff of a One Million Dollar Gene Therapy

      According to a recent article in Archive Today, an international band of amateur biologists, or "biohackers" announced that they intend to eventually market a knock-off of Glybera, a one…

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