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…
KMT2A rearrangements—a DNA fault—lead to treatment-resistant acute lymphoblastic leukemia (ALL) in infants. While around 50% of children with KMT2A rearrangements respond well to intensive chemotherapy, the remaining 50% do…
You caught me: I'm a Swiftie. I first saw Taylor Swift on her Speak Now tour and, in 2 weeks, am taking on the Eras tour in Philadelphia (PS: if you have any…
There is no cure for cystic fibrosis. That is the reason the Cystic Fibrosis Research Institute (CFRI) is very vocal and makes it clear that cooperation is needed from the…
Throughout the globe, there are many countries in which rare disease awareness and support are still lacking. While India has made some strides in improving care for people within…
According to a story from Healio, a recent study found that patients with metastatic pancreatic cancer (more specifically, pancreatic ductal adenocarcinoma, the most common form) that were being treated with…
Over the past few years, more research has been done into the health impacts of breast implants. In fact, researchers and doctors have even coined the term “breast implant illness,”…
The London Marathon took place on April 23, 2023—and one mom was running for an especially important cause: to raise Angelman syndrome awareness. According to an article from the BBC,…
When Kristen Moore’s son Jackson was first diagnosed with tuberous sclerosis complex (TSC), she decided to translate her emotions into action. As WSOC-TV reports, she dove into education, awareness,…
According to a story from Healio, the US Food and Drug Administration (FDA) has recently updated the indication for the drug teprotumumab (marketed as Tepezza). Approved for the treatment of…
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…
Right now, there are a number of treatment options available for mantle cell lymphoma (MCL): chemotherapy (with/without immunotherapy), stem cell transplants, and targeted therapy. Yet despite these options, this…
Lyme disease is an illness caused by Borrelia burgdorferi (B. burgdorferi) bacteria that is transmitted through the bite of an infected deer or western blacklegged tick. If you live…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
May 7, 2023 will be recognized as Cystinosis Awareness Day. This will be a day to spread awareness about the rare disease cystinosis among the general public and the medical…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
Currently, there are no cures for amyotrophic lateral sclerosis (ALS). Symptomatic therapy, alongside disease-modifying treatments like Rilutek, Radicava, and Relyvrio, may be used to manage this condition. However, there…
Collaboration has always made the world go round - and it's no different in rare disease research. Sometimes, the community affected by a certain rare disease is small, which…
On April 26th, I had the honor of speaking at the American Brain Foundation’s Commitment to Cures fundraising dinner during the American Academy of Neurology annual convention in Boston. I…
After becoming a de facto football star while playing at LSU, catching 52 passes for a total 629 yards, tight end (TE) Foster Moreau was drafted by the Las…
When it comes to staying active, 8-year-old R.J. Walters has it covered. His favorite sport right now is basketball. But that hasn’t stopped R.J. from getting his yellow belt in…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
In July 2022, Patient Worthy reported on data from the Phase 3 REAL 4 clinical trial. During the trial, researchers compared the safety, efficacy, and tolerability of Sogroya (somapacitan-beco)…
Dear fellow rare community, It is with great joy that I share my journey of self-discovery and self-love with you. It took me a while to fully embrace my uniqueness…
As bluebird bio ("bluebird") waited on FDA feedback regarding the manufacturing process for lovotibeglogene autotemcel (lovo-cel) genetherapy, the company disclosed that it would most likely miss its Q1 submission…
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