April 21st is AML World Awareness Day! Acute myeloid leukemia (AML) is a rare cancer which affects people around the world. On awareness day, patients, caregivers, healthcare organizations, advocates, physicians,…
Every year almost half of the fourteen thousand patients who have received bone marrow or stem cell transplants will develop chronic graft-versus-host disease (cGVHD). Currently, there are no options…
Acute myeloid leukemia (AML) is a cancer of the bone marrow and blood. It spreads rapidly. Therefore precise and swift treatment is critical. In a recent interview with Medical Life…
Six years ago, researchers began developing single-domain antibodies (nanobodies) to help identify different species of Ebola; their work can be found published in ACS Infectious Diseases. Initially, the nanobodies were created to…
In 2020, Tyrese Dibba wanted to make a change for those who were Deaf and hard-of-hearing. Tyrese himself is Deaf and also has partial vision loss, resulting from his CHARGE…
In the past, there have been a few case reports and other studies which theorized a potential link between eosinophilic conditions, such as eosinophilic esophagitis (EoE), and Celiac disease, a…
According to a recent article, the biopharmaceutical company Amryt has announced a study looking at Mycapssa to treat patients with carcinoid symptoms as a result of neuroendocrine tumors (NET). Neuroendocrine…
According to a recent article, researchers at the WORLDSymposium revealed they are looking into using pentosan polysulfate sodium to treat Maroteaux-Lamy syndrome. MPS VI (Maroteaux-Lamy Syndrome) Maroteaux-Lamy syndrome is…
According to a recent press release from Respira Therapeutics, their investigational treatment for pulmonary arterial hypertension (PAH), RT234, has just been administered to the first patient in a phase 2b…
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…
The American Association for Cancer Research (AACR) Annual Meeting took place from April 8th until the 13th, with lots of interesting data and innovations presented by some of the leaders…
One of the largest problems that stands in the way of rare disease research is funding. It can be difficult to raise the enormous amounts of money needed to plan,…
Two years ago, China approved ensartinib as a second-line therapy for those with ALK+ non-small cell lung cancer (NSCLC). There have been advances over time. Now, according to a late…
A team of researchers from Yale School of Public Health, including Dr. Peter Krause, Dr. Durland Fish, Demerise Johnston, and others, have published a study in the journal Clinical Infectious Diseases documenting a…
Nelson Ambrogio had always looked up to his older brother, Daniel. He has many positive memories of growing up with him. They both shared a passion for soccer, and Nelson…
In the Phase 3 KEYNOTE-522 clinical trial, researchers found that pembrolizumab was effective in treating patients with triple-negative breast cancer (TNBC). According to Medical XPress, researchers wanted to determine whether…
According to a recent article from WFMZ+, the FDA has recently approved Regeneron's supplemental Biologics License Application (sBLA) for Dupixent, granting it priority review. Dupixent, also known by the generic…
Did you know that an estimated 50% of those with lupus have neuropsychiatric symptoms, or symptoms which impact their mental and psychological health and functioning? For example, some people with…
On April 6, 2022, biotechnology company Dialectic Therapeutics announced via press release that its therapeutic candidate DT2216, designed for relapsed or refractory (R/R) cutaneous T-cell lymphoma (CTCL) or peripheral T-cell…
In the United States, Orphan Drug designation is granted by the FDA to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions. “Rare” is defined as…
According to a recent article, a study has found that denosumab therapy leads to patients with osteoporosis having a higher risk of getting osteonecrosis of the jaw in comparison to…
According to a recent article, investigators have proposed a checklist that will aid in determining the strength of potential gene-disease relationships for rare disease. The Need It has already…
April 15, 2022 marks the 9th annual International Pompe Day, designed to help raise awareness and education around Pompe disease. The slogan of International Pompe Day is "Together We…
According to a recent press release, the FDA has recently granted approval to Vijoice - also known under the generic name alpelisib - for the treatment of PIK3CA-related overgrowth spectrum…
A study recently published in the journal ESMO Open has documented new biomarkers which may be able to predict efficacy outcomes of antiangiogenic therapies (such as surufatinib) in patients diagnosed with neuroendocrine…
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