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.
Alzheimer’s disease is the primary cause of the progressive decline of cognitive function in the elderly. It is characterized by underlying complications in the central nervous system (CNS). To date,…
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Researchers Close in on Alzheimer’s Disease As FDA Grants Fast Track Designation to LX1001
The joy of watching your child grow and meet developmental milestones is not always granted to some parents. Clare’s mother, Allison, described her emotions to CHOP after noticing that Clare…
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Treating Rett Syndrome: Clare’s Rare Patient Story
For over thirty years, researchers at the University of Pittsburgh (UPMC) have been observing cases of limited scleroderma, a connective tissue disorder that causes thick hard patches of skin. Scleroderma…
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Researchers Find That Scleroderma Management Should Include Th/To Antibody Testing
[Cancer Management and Research](cancer-management-and-research-journal) » [Volume 14](cancer-management-and-research-archive55-v1655) Feasibility and Safety of Anlotinib Monotherapy for Patients with Previously Treated Advanced Esophageal Squamous Cell Carcinoma: A Real-World Exploratory Study Authors [Zhang S](author_profile.php?id=1654738)…
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Feasibility and Safety of Anlotinib Monotherapy for Patients with Previously Treated Advanced Esophageal Squamous Cell Carcinoma: A Real-World Exploratory Study
Fibrodysplasia ossificans progressiva (FOP) brings a serious meaning to a commonly-used phrase: ‘My big toe.’ According to a recent article in the Orphanet Journal of Rare Diseases, FOP is an…
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FOP: An Abnormality in the Toe is Actually An Ultra-Rare Disease
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It is believed that in 1916 a German doctor named Brachmann first identified a patient with symptoms of a disease later known as Cornelia de Lange syndrome. Then in…
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Cornelia de Lange (CDLS) Syndrome Awareness day is May 14th and Support is Needed Now More Than Ever
A recent article in Labroots stated that the majority of patients who have preleukemic disorders may not be candidates for leukemia. Estimates are that there are about ten to fifteen…
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An Update on Myelodysplastic Syndrome, Flow Cytometry, and Risk of Leukemia
Aeterna Zentaris’s ongoing DETECT trial is evaluating the drug macimorelin (tradename Macrilen™) for childhood-onset growth hormone deficiency (CGHD). As recently announced in an article published by GlobeNewsWire, the study (AEZS-130-PO2),…
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Phase 3 Childhood-Onset Growth Hormone Deficiency Trial Continues Enrollment
Imagine the highs and lows that accompany the doctor’s announcement that your sweet, innocent two-year-old daughter has leukemia. The good news is that your daughter has the type of leukemia…
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A Mother’s Thoughts When Her Two-Year-Old Daughter Was Diagnosed with Acute Lymphoblastic Leukemia
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As featured recently in the Cancer Therapy Advisor, a study has been conducted on patients diagnosed with metastatic renal cell carcinoma (mRCC), who also have elevated inflammation, and sarcopenia. The…
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Study of Patients With Metastatic Renal Cell Carcinoma and Sarcopenia Finds Reduced Survival Following Cytoreductive Nephrectomy
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Al Lupiano, an environmental scientist, recently spoke with Meg Baker of CBS2. Al told her that twenty years ago, he was diagnosed with a brain tumor. The memory and…
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Former Resident of Woodbridge, New Jersey Calls for Action After Learning of Sixty-Five Rare Brain Tumors Linked to Local High School
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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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Compassion Corner: Terminal Cancer Patients Offer Their View of Compassion and Empathy Versus Sympathy
According to a recent article in Genetic Engineering and Biology News, Washington University and University of Hawaii researchers have uncovered a possible drug development candidate in soil bacterium called…
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Researchers Discover Potential New Rare Cancer Drug in Soil Bacterium
Dr. Christopher Austin, a National Institute of Health Director for Transitional Sciences, spoke at last year’s virtual conference honoring Rare Disease Day. According to an article in the NIH Record, Dr.…
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Advocates Ask that the Same Effort Used for COVID Drug Discovery Be Used for Rare Diseases
Korea began its use of effective therapies for idiopathic pulmonary arterial hypertension (IPAH) early in 2000. Idiopathic hypertension has no apparent cause. Yet there have been very few studies assessing…
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Study Identifies People with Idiopathic Pulmonary Arterial Hypertension at Greatest Risk of Death
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Maria’s message is: “when in doubt, get a biopsy." Ten years ago, Maria Sylvia noticed a vertical streak on her thumbnail that at the time she thought was “cool." She…
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An Innocent-Looking Shadow on Her Thumbnail Turned Out to be Subungual Melanoma
Necroptosis, a type of regulated cell death, has been implicated in diseases such as inflammatory bowel disease, as well as neurodegenerative diseases. According to a recent article in News Medical…
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Researchers Find a Path Forward for Patients With Polymyositis
Rise is a nonprofit organization that gives support to the disabled. Its services include rides by Lyft, the popular ride-sharing company. The funds are then reimbursed by their riders’ Medicaid…
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Meningitis: New Program Gives Rides to Work for People with Disabilities
According to information from the National Institute of Health, one in nine elderly people in the U.S. will be affected by Alzheimer’s disease (AD). A recent article in the Globe Newswire…
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LEXEO Therapeutics Announces Positive Clinical Trial Data in Alzheimer’s Disease
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Every year almost half of the fourteen thousand patients who have received bone marrow or stem cell transplants will develop chronic graft-versus-host disease (cGVHD). Currently, there are no options…
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Thousands of Children are Waiting for FDA’s Approval of IMBRUVICA for Pediatric Graft-Versus-Host Disease
Acute myeloid leukemia (AML) is a cancer of the bone marrow and blood. It spreads rapidly. Therefore precise and swift treatment is critical. In a recent interview with Medical Life…
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AML Took Her Beloved Three-Year-Old Son, Now She’s Determined to Strike Back
If you have not heard of craniosynostosis, it is understandable, as the disorder affects only one infant out of approximately 2500. A recent article published in the Washington News…
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Craniosynostosis: An Operation Saved His Brain from Being Crushed
Professors Susan Treves and Francesco Zorzato, Basel University researchers, have recently presented the first potential approach to congenital muscle diseases. Their report was published in a recent article by Science…
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Two Professors Develop An Approach to Treat Congenital Myopathy
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In 1873, a Russian physician coined the term “multiple myeloma” after discovering a patient with eight different forms of bone marrow tumors. In the early years, patients were treated with…
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New Treatment Options Have Doubled Survival in Multiple Myeloma
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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…
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Compassion Corner: A Doctor’s Compassion Saved Her Son’s Life