The FDA has recently approved a treatment called Pyrukynd as a therapeutic option for hemolytic anemia disorder sparked by pyruvate kinase deficiency. Hemolytic anemia is a condition where red blood…
A recent study examined the impact of age on clinical outcomes for patients diagnosed with chronic lymphocytic leukemia (CLL). This study was published in the journal Blood Advances. All patients in…
Written By Joslyn Crowe Caring for someone with a rare disease, especially a child or a family member, can be a difficult and emotional experience. Many people become a caregiver…
Silmitasertib, an investigative therapy for biliary tract cancer (BTC), has been awarded Orphan Drug Designation by the FDA. Researchers and the regulatory agency are hopeful that this therapy could become…
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…
Written by Matt Horsnell, Community Manager, TREND Community TREND Community is a leader in evidence acceleration through social data exploration for rare and chronic illnesses. Patient Worthy is at the forefront…
A recent study has uncovered that a CAR-T therapy called Kymriah could lead to improved results for follicular lymphoma patients. Specifically, it was found to be both effective and safe…
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…
Continued From Part One Case #2 The second case involved a sixteen-year-old male in the ICU unit with suspected bacterial hypoxic respiratory failure associated with viral pneumonia. The teen’s history…
Claire Barrow is a 14 year old CEO, entrepreneur, rare disease patient, and advocate for the rare disease community. She is the founder and CEO of an app called RareGuru which…
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…
According to a study published in PLOS Medicine, only around half of pragmatic or 'real-world' trials collect data as reported by patients or involve patients as partners in research. This…
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…
A recent study has demonstrated that neuromyelitis optica spectrum disorder (NMSOD) patients treated with eculizumab have a lower risk of adjudicated relapse compared to those treated with placebo. This study…
A Phase 3 clinical trial for advanced biliary tract cancer (BTC) has found that there is an improved overall survival rate when patients are given the immunotherapy durvalumab in combination…
PTC Therapeutics Inc. has just announced that their supplemental new drug application for Evrysdi (risdiplam) has been given priority review by the FDA. The aim is to expand the indication…
A recent study published in Advances in Rheumatology has uncovered 2 potential biomarkers which could be used to predict which lupus nephritis (LN) patients are at the highest risk of severe renal…
A study recently published in the American Journal of Transplantation evaluated whether or not transplanting the kidney of a pig could be a viable and safe options for human patients. This study…
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…
According to a recent article, patients diagnosed with minimal change disease are found to have autoantibodies targeting nephrin, which supports previous research. Minimal Change Disease (MCD) Minimal change disease is…
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…
According to a recent article, researchers were able to produce the first images of an enzyme that may figure out the unknown cause of a rare group of hereditary neurodegenerative…
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 Pulmonary Hypertension Association recently announced it is offering junior investigators who are interested in pulmonary arterial hypertension (PAH) a two-year grant. Pulmonary Arterial Hypertension (PAH) Pulmonary arterial hypertension (PAH)…
Avrobio has just announced that they will be stopping work on their investigative gene therapy for Fabry disease based on unexpected and disappointing results from a Phase 2 clinical trial.…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
