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.
source: pixabay.com
Arrowhead Pharmaceuticals recently announced positive twenty-four-week biopsy results from four patients who participated in the first cohort of the Phase II clinical trial of ARO-AAT. ARO-AAT is Arrowhead’s investigational…
Continue Reading
Patients with Alpha-1 Antitrypsin Deficiency Liver Disease Show Improvement After Six Months of Experimental Treatment
source: pixabay.com
Dr. Naveen Pemmaraju recently interviewed with Targeted Oncology and discussed the many advances in myeloproliferative neoplasms (MPNs) treatment as well as the role of social media during the pandemic.…
Continue Reading
ICYMI: Updates on Myeloproliferative Neoplasms
source: pixabay.com
According to a recent article in Business Wire, it is estimated that five million people in the United States over the age of sixty-five are coping with Alzheimer’s at…
Continue Reading
Patients Receive First Dosing of Experimental Drug Targeting Neurodegenerative Diseases
source: pixabay.com
Dr. Richard Furie at New York’s Feinstein Institutes recently interviewed with MedPage Today. Dr. Furie discussed the prognosis for lupus nephritis and belimumab (Benlysta), a recently-tested drug that appears…
Continue Reading
Belimumab is the Second Drug to be Developed for Lupus in Over Fifty Years
Several decades ago, gene therapy seemed to be on target to treat a disease by simply replacing a defective gene with a healthy one. A recent article republished in APNews…
Continue Reading
CRISPR, The “Genetic Surgeon”
According to a recent article in MedicalXpress, in 2004 Professor Sophie Lucas of the Louvain de Duve Institute began studying immunosuppressive cells that are known to block immune responses in…
Continue Reading
Testing of Immunosuppressive Cells Leads to Tumor Regression
An article that appeared recently in Psychology Today asks whether dementia is responsible for falls. The answer brings to mind the many medical and environmental factors that affect the elderly…
Continue Reading
Dementia Plus Medical and Environmental Factors Often Result in Falls
According to a recent article in Genetic Engineering and Biotechnology News, the neural circuit that is responsible for insomnia has been identified by scientists at the Cold Spring Harbor Laboratory…
Continue Reading
Cold Spring Harbor Scientists Identify Neural Circuit That Causes Insomnia
As reported in Science Daily, researchers at the University of Bristol’s MRC Epidemiology Unit working with pharmaceutical companies have developed an approach to reduce the failure rate of developing drugs.…
Continue Reading
Scientists Develop a New Approach That Reduces the Failure Rate In Drug Development
According to a recent article in MedicalXpress, researchers in Japan led by Dr. Shinsuke Ishigaki and his team at Nagoya University, have identified new molecular details about tau’s activity. Alzheimer’s,…
Continue Reading
Researchers at Nagoya University are One Step Closer to Unraveling the Tau Mystery in Neurodegenerative Diseases
source: pixabay.com
Elliot Preddie, age 12, is ready to get back to his favorite sport, football. He had to put off playing due to sickle cell disease (SCD). Elliot substituted taekwondo and…
Continue Reading
Child With Sickle Cell Disease Received Successful Stem Cell Transplant
Praise Aki Amedal, a Nigerian living in the city of Oriade, has just completed his first book entitled The Biological Prisoner . . . Finding Hope in a Failing Body.…
Continue Reading
A Young Nigerian With a Rare Disease Has Written a Book About His Search for a Cure
source: pixabay.com
Phase III clinical trial results of the triple therapy combination for the treatment of severe aplastic anemia (SAA) were set forth in a recent News Medical Life Sciences article.…
Continue Reading
Severe Aplastic Anemia Patients’ Response Rates Improved with a New Triple Therapy
Ariella Pacheco of Encinitas, California is a 17-year-old in her senior year at a Carmel Valley Catholic School. Ariella spoke to a reporter for MSN about her new-found passion to…
Continue Reading
Special Dolls for Children With Rare Diseases
Business Insider recently interviewed Marissa Oliver, a resident of New York City, who told us about the problems she has encountered during her recovery from COVID-19. Marissa, an arts manager,…
Continue Reading
Researchers Are Investigating a Possible Connection Between Chronic Fatigue Syndrome and COVID-19
A growing wait list is creating additional stress on patients needing organ transplants. These patients are increasingly desperate according to an article recently published in Genetic Engineering & Biotechnology News.…
Continue Reading
One Answer to the Organ Donor Shortage: A Drug That Revitalizes Older Cells
A recent article in Med City News outlines the difficult choices patients must make regarding immune compromising but life-saving therapy during the 2020 pandemic. Do they postpone their treatment or…
Continue Reading
Early Stages of Precision Medicine Being Developed for Cancer Patients During the COVID-19 Pandemic
According to a recent article in Genetic Engineering & Biotechnology News, researchers at the UofSC have presented data stating that the cannabis compound tetrahydrocannabinol (THC) is effective against respiratory distress syndrome…
Continue Reading
Researchers Are Studying the Use of THC For the Treatment of ARDS caused by COVID-19
A recent article in the Korea Biomed Review reported that the joint research effort by Seoul National Medical College and Helixmith of San Diego, California, found Helixmith’s VM507 antibody effective…
Continue Reading
Joint Efforts by Korean and California Research Teams Develop A Drug That Restores Renal Intervascular Cell Function
Professor Illana Gozes and her team at Tel Aviv University recently developed NAP (CP201). It is an experimental drug that has been proven to be effective in the treatment of…
Continue Reading
An Experimental Drug for Alzheimer’s is Being Tested to Treat Autism
Salk Institute scientists have scored a significant gain in their quest to find a functional treatment for type 1 diabetes. According to an article in Salk News, scientists at the…
Continue Reading
Salk Institute Team Develops the First Insulin-Producing Human Cells That Control Blood Sugar in Type 1 Diabetes
According to a recent article in FierceBio, Andrea Mazzocchi, Known Medicine CEO, is not trying to reinvent the wheel. On the contrary, her company will be providing a tool to…
Continue Reading
This Startup is Determined to Take the Guesswork Out of Personalized Treatments for Cancer
According to an article in the publication Labroots, autism spectrum disorder (ASD) may be the result of interaction between environmental and genetic factors. For instance, researchers point out that ASD…
Continue Reading
Scientists Find an Array of Genes That May Lead to Precision Therapies for Various Types of Autism
Huda Zoghni was born in Lebanon. Huda attended medical school at the American University located in Beirut in 1975. According to an article in Discover, Huda was forced to leave…
Continue Reading
She Survived the Lebanese Civil War and 30 Years of Challenges to Help Rett Syndrome Patients
The winning entry for the 2019 Student Voice was published recently in the Orphanet Journal of Rare Diseases. The prize-winning article, a warm and personal account, was written by Annie…
Continue Reading
A Med Student Assignment Became a Valuable Lesson
