PNH Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease where blood cells break apart and cause the contents to circulate freely within the bloodstream. This condition is caused by a…
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 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…
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 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…
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…
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…
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…
Elizabeth Dravis is a PhD student studying public health, a medical professional, a runner, and a mother. She also is a minimal change disease (MCD) patient. This is her diagnosis…
Definitive Healthcare launched a survey in November 2021 to help physicians, researchers, and the rare community understand what the biggest challenges are for providing a rare disease diagnosis and treating…
Myrtelle Inc. has just announced that they have been granted three new designations from the FDA for their investigative gene therapy called rAAV-Olig001-ASPA. This therapy is being investigated as a…
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…
Juvena Therapeutics has just announced that they've received a new grant which will fund their research of myotonic dystrophy type 1 (DM1). This was a very competitive grant which totals…
An article recently highlighted the stories of young patients with Ehlers-Danlos syndrome and how they are taking their diagnoses to spearhead research about the rare disease. Ehlers-Danlos Syndrome (EDS) Ehlers-Danlos…
The COVID era emphasized the importance of maintaining a strong immune system. But what about people who are less fortunate? Johns Hopkins researchers recently announced some good news for…
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…
Patient Worthy partner HCU Network America and RARE X, a rare disease patient data service organization, have recently partnered in order to create a registry of patients living with homocystinuria,…
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 poster recently presented at the South Beach Symposium Medical Dermatology Summit by Dr. Brandon Kirsch documented that sofpironium bromide gel topical treatment can reduce how severe primary axillary hyperhidrosis…
According to a case-based roundtable meeting for Targeted Oncology, Matthew A. Lunning, DO, talked about second-line therapies for diffuse large B-cell lymphoma and the results of a study called L-MIND.…
UCB Inc., a biopharmaceutical company, has announced positive results for its RAISE Phase 3 trial (NCT04115293) investigating zilucoplan against placebo. According to a recent article in Biospace, zilucoplan met…
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…
This story is brought to you by our friends at HAE Junior in the Czech Republic. I think that the best gift is one that will please not only the…
A study recently published in the journal Blood Advances has demonstrated that brentuximab vedotin (BV) treatment leads to improved outcomes for primary cutaneous anapestic large-cell lymphoma (C-ALCL) and CD30-expressing mycosis fungoides (MF)…
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…
© Copyright 2024 Patient Worthy
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.
