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.
Professor Miranda Orr of Winston Salem’s School of Medicine in North Carolina leads a team of investigators who have detected senescent cells in patients with Alzheimer’s disease. These cells, found…
Continue ReadingA Potential New Target for Alzheimer’s Called Zombie Cells
The World Health Organization (the WHO) announced World Leprosy Day which this year will be celebrated on January 30th. The theme of the celebration is “United for Dignity.” It…
Continue ReadingJanuary 30th is World Leprosy Day: Let Us Be United for Dignity
Julia Lefelar, a resident of Gaithersburg, MD, was telling a friend about her years of seeing different doctors trying to find the cause of her illness. After a while, her…
Continue ReadingAfter Being Examined By Doctors for 17 Years, Julia Finally Received an Accurate MOG Diagnosis
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…
Continue ReadingCompassion Corner: A Patient Almost Died Because of a Doctor’s Lack of Understanding and Compassion
The Critical Path Institute (C-Path), formed in 2005, is a nonprofit, independent (public/private) partnership. The Institute evolved from the FDA’s 2004 White Paper known as the Critical Path Report and…
Early detection is the key to treating so many types of cancer. Autoimmune hepatitis (AIH), chronic inflammation of the liver, is brought about by an immune system disorder. AIH must…
An article in PubMed dated October 8, 2021 outlines immune therapies treating myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) patients. Both cancers are hematologic malignancies that begin in the…
Continue ReadingRecent Advances in Acute Myeloid Leukemia and Myelodysplastic Syndromes
BeyondSpring Pharmaceuticals has issued a press release announcing that the FDA presented the company with a complete response letter pertaining to its drug plinabulin and granulocyte colony-stimulating factor (G-CSF). The…
Continue ReadingThe Long Road to FDA Approval of Plinabulin and G-CSF to Prevent Chemotherapy Induced Neutropenia
This week the Guardian reported that a pig’s heart, which was genetically modified, had been transplanted into a patient by doctors who were trying to save his life. The patient was…
Continue ReadingUniversity of Maryland Doctors Transplant a Modified Pig’s Heart In a Human Patient
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…
Continue ReadingCompassion Corner: Doing What is Best For the Patient May Override a Doctor’s Compassion
Biogen Inc. recently announced data that included extended results from its earlier Phase 3b NOVA study on the administration of natalizumab and the evaluation of the drugs Tysabri® and Vumerity. …
Continue ReadingBiogen Announces New Data From Its Portfolio of Treatments for Multiple Sclerosis (MS)
Diagnosing amyloidosis (abnormal protein in tissue) is a complex process. Over eighty percent of amyloidosis patients are undiagnosed. The disease takes hold after the blood plasma cells in the bone…
Continue ReadingAttralus is Developing a New Treatment for Systemic Amyloidosis
Nonalcoholic steatohepatitis is a liver disease, causing fat to build up in the liver. The resulting inflammation may lead to liver cancer or cirrhosis (scarring) and possible liver failure. As…
Continue ReadingKorean Group’s Successful Experiment to Treat Nonalcoholic Steatohepatitis
Many people living in rural areas of the U.S. must drive a long distance to receive their medical treatments. According to Yahoo News, twenty-nine-year-old Leah Stavenhagen, now living and…
Continue ReadingDiagnosed with ALS at Age 29, Leah Advocates to Increase Funding for Research
The recent FDA approval of Vyvgart (efgartigimod) for generalized myasthenia gravis (gMG) marks the first drug approval for Argenx. Vyvgart is designed specifically for adults who test positive for…
Continue ReadingICYMI: First FDA Approval for Generalized Myasthenia Gravis
WKBW Buffalo recently interviewed ALS patients who are advocating for a bill that would grant access to clinical trial drugs during Phase III and before they go through the…
Continue ReadingALS Patients Ask Congress to Pass a Bill Allowing Access to Unapproved Drugs
Continued From Part One 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.…
Continue ReadingCompassion Corner: Compassionate Use, Expanded Access Part Two: The Terminology Is Different But the Unmet Need Is the Same
ANCA-associated vasculitis (AAV) generally targets two enzymes, myeloperoxidase (MPO) or proteinase 3 (PR3) located in the granules of monocytes and neutrophils. These enzymes play a critical role in the…
Continue Reading Two Recent FDA and EMA Approvals Bring Hope to ANCA Associated-Vasculitis Patients
Catherine Nettles Cutter, a professor at Penn State University, vividly recalls two years of her life spent trying to recover from a bodysurfing accident that resulted in the mysterious loss…
Continue ReadingA Body Surfing Accident Led to Eagle Syndrome and Rotational Vertebral Artery Syndrome
One 66-year-old patient who lives in Poughkeepsie, N.Y. received the same diagnosis from three doctors. Each doctor suggested that his symptoms pointed to temporomandibular (TMJ) joint pain. The patient and…
Continue ReadingThe Doctors Said TMJ, But the Patient Said Giant Cell Arteritis
After two years of in vitro fertilization treatments, Amber Freed, Age 38, and her husband Mark Freed of Denver, Colorado welcomed their twin babies Riley and Maxwell into the world.…
Continue ReadingA Mother Has Raised Millions to Develop a New Drug to Treat SLC6A1
Victoria Gray’s recent interview with NPR confirmed that her genetically modified blood cells have truly transformed her life. Victoria volunteered in the first-ever attempt in the U.S. to use CRISPR,…
Continue ReadingICYMI: The First Patient to Receive CRISPR for Sickle Cell Disease
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…
Continue ReadingCompassion Corner: Compassionate Use, Expanded Access Part One: The Terminology Is Different But the Unmet Need Is the Same
After several clinical setbacks, Vertex Pharmaceuticals found promising results in its study of a drug to treat a genetic kidney disease called focal segmental glomerulosclerosis (FSGS). As reported recently by…
Continue ReadingA New Drug to Treat Focal Segmental Glomerulosclerosis and Renewed Hope for Vertex Pharmaceuticals
A recent article published in Vascular News highlights the importance of plasma desmosine, an elastin component, that provides elasticity for tissue. A breakdown of elastin leads to aortic dilatation and…
Continue ReadingBritish Heart Foundation Study Identifies Acute Aortic Syndromes
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