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.
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: Compassion Should be Reciprocal: The Rising Suicide Rate of Physicians and Nurses
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…
Continue ReadingNon-Alcoholic Fatty Liver Disease (NAFLD) in Pregnancy has Nearly Tripled in the Past Ten Years
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…
Continue ReadingSarepta’s Duchenne Gene Therapy Drug Cleared for Accelerated Approval
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…
Continue ReadingPositive Interim Findings in Alzheimer’s Clinical Trial
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…
Continue ReadingCanada Will Provide $1.5 Billion to Provinces to Support Rare Disease Programs
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,…
Continue ReadingAdvanced Leukemia: Test of Oral Revumenib Brought Impressive Results
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…
Continue ReadingIncreasing Patient Engagement in Clinical Trials
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…
Continue ReadingOpinion: The Urgency to Address the Issue of Rare Diseases in India
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…
Continue ReadingA New Medicine for ALS Patients with the SOD1 Mutation
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…
Continue ReadingMay is Cystic Fibrosis (CF) Month and Some Patients Are Living Past Middle Age Due to Treatment Advances
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: An Honest Account by an Orthopedic Surgeon Who Found the Key to Compassion
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…
Continue ReadingA Rare Look at the Origins of Genetic Disorders: PI3K Delta Syndrome 2
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…
Continue ReadingHow Can We Tell What Mental Health Tech is Actually Effective?
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…
Continue ReadingTheir Child Has SYNGAP1 Syndrome, Causing the Family to Face Overwhelming Challenges
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…
Continue ReadingA New National Analysis Indicates that Higher Levels of Black Primary Care Physicians Means Longer Life Expectancy for Black Patients
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…
Continue ReadingBrainStorm Files an ALS Drug Application Despite the FDA’s Protest
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…
Continue ReadingAmyotrophic Lateral Sclerosis Drug Developers Set Their Sights on a New Treatment Target
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…
Continue ReadingFor Patients Under Consideration for CAR-T Therapy The Cancer Support Community/Gilda’s Club Has A Free Program
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…
Continue ReadingThe Chan Zuckerberg Initiative is Providing Financial Support for Patient-Centered Rare Disease Research
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…
Continue ReadingAmpreloxetine Failed the First Time but Emerged a Winner for Multiple System Atrophy
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…
Continue ReadingFDA Approves New Treatment for Rare Cancer PEComa
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…
Continue ReadingA Research Group Finds Most Ovarian Cancers Begin in the Fallopian Tubes. Should They Be Removed?
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…
Continue ReadingMutual Trust Between Doctors and Parents of Children with Rare Diseases Means Optimum Care for the Child
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