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.
The Chow family was told that their 33-year-old daughter, Brittney, would have to wait about twelve years for a kidney transplant. There were currently no kidneys available for transplantation. Elizabeth…
In March the FDA’s expert advisers voted 11 to 2 in favor of approving Roche’s antibody drug Polivy to treat lymphoma patients. According to an article in BiopharmaDive, the decision…
The safety, pharmacokinetics (absorption), and tolerability of the investigational eIF2B activator ABBV-CLS-7262 is being evaluated in a Phase 1b clinical study to treat patients with Vanishing White Matter (VWM) Disease.…
PhRMA Public Affairs recently carried the good news that Governor Holcomb of Indiana has joined the governors of Arkansas and West Virginia by signing a law aptly named 'Share the…
This week PR Newswire carried an announcement by the biotechnology company Immunic Inc. of preclinical data confirming that Vidofludimus Calcium (IMU 838) may be a Nurr 1-Activator reinforcement. Immunic is…
For patients who were given a diagnosis of advanced, inoperable (unresectable) biliary tract cancer, an article appeared in Healio with some encouraging news. Gemcitabine and cisplatin drugs have…
Omisirge (omidubicel) is the first allogeneic (from a donor) product for SCT that has been the recipient of FDA approval. The FDA's nod moves Omisirge into the realm of…
The damage caused by cocaine can sometimes be misdiagnosed as a rare disease: granulomatosis with polyangiitis. Cocaine use has been increasing in America and the UK, resulting in serious…
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 the last decade, NAFLD in pregnancy has nearly tripled in concert with the worldwide increase in diabetes. Mayo Clinic reports that NAFLD in general affects one-fourth of the…
Gene therapy has seemed out of reach for decades. Yet advocates have considered it a way to have damaged or missing genes restore dystrophin and other proteins. Doctors and advocates…
A Business Wire Press Release on April 26, 2023, announced positive interim clinical data on an investigational RNAi therapeutic. Alnylam Pharmaceuticals and its partner Regeneron Pharmaceuticals heralded interim results…
Canada’s unemployment rate is at a record low. Industries have recovered 126% of the jobs lost by the pandemic. On the health front, scientific knowledge and the ability to…
For patients with refractory or relapsed KMT2A rearrangement or NPM1-mutations, the AUGMENT 101 study of the investigational menin inhibitor revumenib yielded impressive results. According to an article in MedicalXpress,…
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…
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