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.
Clinical trials are often mentioned as an afterthought when discussing the length of time it takes for drugs to be approved by the FDA. On the contrary, clinical trials are…
Prasanna Shirol, Executive Director of the Organization for Rare Diseases India (ORDI), expresses his opinion to the Times of India about the urgency to address the issue of rare…
The FDA estimates that there are about five hundred people in the USA with the SOD1 mutation out of a total of approximately thirty thousand ALS patients. According to a…
There is no cure for cystic fibrosis. That is the reason the Cystic Fibrosis Research Institute (CFRI) is very vocal and makes it clear that cooperation is needed from the…
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…
People with rare undiagnosed conditions present challenges to doctors when they are attempting to understand the root causes of their patient’s health problems. A recent article in Medical Xpress reports…
STAT NEWS recently published an op-ed with a provocative question: How do we tell the difference between legitimate mental health tech and snake oil? Looking back to the early…
Perhaps one of the most difficult challenges of families impacted by certain rare diseases is trying to appear “normal” for the sake of others in the family. That is one…
Over the years, there has been minimal improvement in the life expectancy differences between white and black people living in the United States. Yet, according to an article featured…
The FDA has carried on a year-long disagreement with BrainStorm Cell Therapies, the developer of the experimental stem cell treatment called NurOwn. It is referred to as a personalized cell…
On March 22, 2023, via PRNewswire, the FDA announced that its CNS Drugs Advisory Committee voted unanimously in favor of a potential accelerated approval of tofersen to treat people with…
No One Should Face Cancer Alone. That is the mission of the non-profit Cancer Support Community (CSC). It offers emotional support and hope via programs designed for cancer patients, cancer…
In 2003 Ricky Safer was diagnosed with primary sclerosing cholangitis (PSC). Her doctors were not able to tell her much about the origin of her disease. Nor were they able…
Theravance Biopharma’s late-stage failure suddenly became a jump-off point for treating multiple system atrophy (MSA) according to a recently published article in Neurology Live. After the failure of two…
Caring is Sharing and Getting Involved. On April 17th Landmarks all over the world will be asked to ‘Light It Red’ in honor of Hemophilia Day. The theme of this…
Discovering a treatment for a rare disease is challenging but it can also be rewarding. This is especially true when treating an extremely rare disease such as perivascular epithelioid…
Ayla Bashir and her parents, Sobia Qureshi and Zahid Bashir, have participated in a medical first with Ayla surviving Pompe disease. The same disease caused the death of her…
The Ovarian Cancer Research Alliance stated in new guidance that evidence indicates the majority of ovarian cancers begin in fallopian tubes. The president of the alliance, Audra Moran, was…
The complexity of rare diseases makes communication between adults involved with the care of these children a vital tool. Communication and mutual trust are of the utmost importance. About the…
Dr. Amish Desai speaks with the utmost sincerity on KevinMD's recent podcast. The doctor explained the difficulty he encountered trying to balance treatment for his father while giving the same quality…
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…
In March of 2021, twenty-eight-year-old Alix Burnard of Newbury, Berkshire UK had several Covid-19 tests due to a suspected infection. All three tests were negative, so she felt it…
Imagine being Rowan Marshall, a seven-year-old first-grade student with a debilitating rare disease. Rowan made it through the new student formalities. He is doing well at school. Now he faces…
Researchers have been grappling for years with the STAT5 protein as a tool to fight cancer. STAT5 is critical because it is partly responsible for the development and progression…
After winning the 1998 100m butterfly in Perth, breaking a world record, winning 54 international medals, and becoming the Swimming Hall of Fame inductee in 2022, Michael Klim appeared…
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