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.
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…
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…
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…
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. …
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…
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…
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…
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…
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…
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.…
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…
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…
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…
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.…
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,…
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…
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…
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…
Healio recently published results of the CheckMate 649 study confirming the benefits of combining nivolumab and chemotherapy in treating newly diagnosed patients with gastroesophageal junction cancer and advanced gastric and…
For many years, scientists have known about toxic clumps forming in patients with Huntington’s disease (HD). However, until recently, scientists relied on examining post mortem brain samples of HD…
According to Dr. Deepak Chellapandian, a Johns Hopkins specialist in bone marrow transplant for rare diseases, the prognosis for his patient was very poor. Jocelyn was born with chronic…
Washington University Researchers readily acknowledge the attributes of sonodynamic therapy (SDT) as a promising therapeutic method to treat hepatocellular carcinoma (HCC). SDT is noninvasive with high tissue penetration, but…
Dr. Richard Stone recently interviewed with Targeted Oncology to discuss challenging acute myeloid leukemia subtypes and a list of available therapies. Dr. Stone was asked to look back at…
Pancreatic cancer so far has been able to escape early detection. When it is discovered, it is usually in an advanced stage and difficult to treat. According to a…
Discovering a treatment for a rare disease is challenging and rewarding, especially when it is an extremely rare disease such as perivascular epithelioid cell tumor (PEComa). Biospace recently carried…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
