Until recently, multiple myeloma patients were reliant upon treatment with immunomodulatory drugs, anti-CD38 antibodies, proteasome inhibitors, and autologous (patient-derived) stem cell transplants. According to a recent article in CancerConnect, patients…
Alveolar capillary dysplasia with misalignment of the pulmonary veins (ACD/MPV), or alveolar capillary dysplasia (ACD) for short, is a rare, congenital, and life-threatening disorder. Without receiving lung transplants in infancy,…
The National Medical Products Administration (NMPA) of China has recently approved surufatinib for drug registration for the treatment of advanced pancreatic neuroendocrine tumors (pNETs). HUTCHMED Limited recently announced the news,…
Transcription factor SOX 11 is encoded by the SOX11 gene. Altogether, this protein is known to play a role in embryonic neurogenesis and tissue formation. However, an estimated 90% of patients with…
Patient Worthy signed up to cover several sessions at the Huntington's Disease Society of America's (HDSA) 36th Annual Convention, which was held virtually this year. This event features informative presentations…
HAE Junior’s drawing exhibition unveiled the dreams, wishes, and ambitions of children & teenagers living with hereditary angioedema (HAE). The patient organization HAE Junior organized a drawing exhibition named We…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Hannah Creel was only 18 months old when she was first diagnosed with cystinosis, a rare disorder that affects all organs in the body, in particular the kidneys. Her parents…
When Taylor Brown was just two years old, her mother TaMetress Reed began noticing certain symptoms, such as hardening and tightening patches of skin. As Brown grew up, the symptoms…
According to a relatively recent news release from biopharmaceutical company Applied Therapeutics, Inc., AT-007 received Fast Track designation from the FDA. This therapy, which has also received both Orphan Drug…
Patient Worthy signed up to cover several sessions at the Huntington's Disease Society of America's (HDSA) 36th Annual Convention, which was held virtually this year. This event features informative presentations…
We're still trying to understand COVID-19, and it may take us years and years to fully comprehend the respiratory virus. A recent case study published in the Canadian Medical Association…
Internationally, over 400,000 people die of malaria each year. In many cases, a majority of these deaths occur in sub-Saharan Africa; children are the most vulnerable. However, according to Medical…
You may know Jessie J from some of her hit singles like "Price Tag" or "Bang Bang," but did you know that she is also a rare disease patient? She…
For the first time, a research study has discovered a genetic marker related to high-risk neuroblastoma. According to Medical XPress, researchers determined that anaplastic lymphoma kinase (ALK) gene mutations and…
As reported by Oncology Nursing News, gunagratinib (ICP-192) was recently granted Orphan Drug designation by the FDA. Altogether, this treatment is being evaluated as a potential treatment for patients…
Patient Worthy signed up to cover several sessions at the Huntington's Disease Society of America's (HDSA) 36th Annual Convention, which was held virtually this year. This event features informative presentations…
In an innovative collaboration, the TAPS Support Foundation and the Fetal Therapy team from the Leiden University Medical Center announced today a new joint initiative – The Twin Talks Webinar…
The pressure was on. Researchers had been studying Alzheimer’s for decades. Every morsel of discovery was hailed as moving closer to a cure and results were always expected soon…
Currently, there are no real treatments for patients with primary mitochondrial myopathy (PMM). Most therapies, such as physical, occupational, or speech therapy, are designed to simply lessen or mitigate symptoms.…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
In late May 2021, the FDA granted accelerated approval to Truseltiq (infigratinib). As reported by Cure, this therapy is designed for adult patients with unresectable locally advanced, previously treated, or…
A recent study presented in early June showcased how, despite some similarities, psoriatic arthritis with axial involvement (axial PsA) patients and axial spondyloarthritis (axSpA) patients have meaningful differences. These findings…
According to a recent article in Biospace, patients with advanced systemic mastocytosis (SM) can turn to a newly FDA-approved treatment by Blueprint Medicines called Ayvakit (avapritinib). The drug is the…
University of Illinois Researchers have uncovered novel information about pulmonary fibrosis. Their research demonstrates that fibrosis in the lungs can be reversed, and tissue can be repaired using microgel-coated mesenchymal…
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