A retrospective study recently published in the journal Pediatric Rheumatology has detailed the different antiphospholipid syndrome (APS) phenotypes in pediatric patients in effort to develop care approaches which are more personalized. APS APS is…
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…
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…
According to a story in the WSJ, the FDA has approved a new therapy for multiple myeloma patients. The therapy is called Carvykti and it is now available as a…
Currently, 95% of rare diseases don't have an effective treatment. Progress is being made, but there is just so much ground to cover in rare disease research. Studying rare diseases…
March is National Kidney Month! This month is about raising awareness for and education of kidney diseases, alongside improving research and outcomes for kidney disease patients. The Polycystic Kidney Disease…
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…
A research study led by Professor Beishi Zheng from Columbia University has studied the immunogenetics of gastrointestinal (GI) cancers. The Study This study demonstrated that individuals who are diagnosed with…
Bonair Daydreams, an inspirational cardline, was founded by a woman who understands human emotions and exactly what words to say in every moment of joy, grief, pain, and hesitance. 27…
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)…
Regeneron Pharmaceuticals has just announced the results from their Phase 2 trial of aflibercept. The trial aimed to compare an 8mg dose of the treatment to a 2mg dose, which…
The FDA has recently announced that they have put a clinical trial for relapsed or refractory T-cell lymphoma patients and cutaneous T-cell lymphoma patients on hold. This trial was a…
The FDA has just announced that they are reevaluating their approvals for Ukoniq. This reevaluation comes after study findings which demonstrated there may be an increased risk of fatality for…
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…
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…
Extensive sweating, or hyperhidrosis, can be an extremely frustrating diagnosis. Although sweating itself is an incredibly normal and healthy process, when it happens excessively, it can impact your day to…
Soleo Health has just announced that they have been provided access to lumasiran by Alnylam Pharmaceuticals. This access will allow the company to administer the treatment to patients diagnosed with…
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…
Alnylam Pharmaceuticals has just announced that they will be starting a Phase 2 trial for their RNAi therapeutic called Lumasiran. This trial will investigate both the safety as well as…
The FDA has just announced that they have granted Orphan Drug Designation to an investigative treatment called eplontersen. This therapy is being studied as a treatment option for transthyretin (ATTR)…
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…
Rare cancers are a unique research area which necessitate specified attention. Cancer Commons, Pattern.org, and the Rare Cancer Research Foundation are three organizations dedicated to improving the lives of those…
© Copyright 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.
