Often, one's own experiences inspire them to advocate for a certain cause. Abbey Hauser is an example, as she has become a voice for young adults within the rare disease…
In August of 2021, Intellia Therapeutics of Cambridge and its partner Regeneron Pharmaceuticals published a study reported in the Boston Globe, showing results of a single treatment using CRISPR-Cas9 technology.…
The American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting took place this year from February 25-28, 2022. During the meeting, stakeholders gathered to discuss new technologies, healthcare reform,…
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…
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…
Walks are a great way to raise awareness and money for a cause, and they're also good for exercising and spending some time with friends and family. Recently, the Southwest…
According to a story from Targeted Oncology, the company Genmab has recently announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation to its investigational therapy…
Since its founding in 2009, The Assistance Fund (TAF), an independent charitable patient assistance organization, has helped patients and families within the rare disease and chronic illness sphere with financial…
Unintended weight loss is a symptom associated with many different forms of cancer. But what can this weight loss tell researchers about the patients and their outcomes? Healio shares a…
An article recently published in Medical Xpress outlined research into memory recall in Rett syndrome patients. Not only did it look into the underlying causes of diminished memory recall, but…
Until recently, patients with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) did not have an FDA-approved treatment option. This all changed when the regulatory agency granted approval to ZTALMY, also…
According to a recent article, although the number of ticks are rising in Germany currently, the cases of tick-borne encephalitis have been surprisingly lower. Researchers found that this is due…
Recently, two medical professionals were interviewed where they discussed their recommendations for the guidelines pertaining to managing psoriatic arthritis. Psoriatic Arthritis Psoriatic arthritis is a type of inflammatory arthritis that…
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,…
ProQR Therapeutics is working to develop unique therapeutic solutions for genetic eye diseases. Currently, the company is evaluating its therapeutic candidate ultevursen within the Phase 2/3 Sirius and Celeste clinical trials; you can read…
Each year, an estimated 11,000 people across the globe are diagnosed with relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL) or lymphoblastic lymphoma (LBL). However, there is only one…
Rare disease research itself is often overlooked in the medical sphere, so it's not difficult to imagine how many singular aspects of rare disease research are forgotten about completely. One…
At first, 34-year-old Chris Mee wasn’t too worried about his back pain. In fact, he kind of brushed it off – thinking it could just be sciatica, or aching from…
When it comes to finding treatments for diseases, whether they're common or rare, the process doesn't always start from scratch. In many cases, existing drugs are taken and studied in…
Over the last few years, there has been much discussion around VLA1553, a Chikungunya virus vaccine candidate. The vaccine has so far earned Fast Track and Breakthrough Therapy designations within…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions. Rare conditions are defined as those affecting…
Written by Sumeet Maniar, CEO, WellBrain Continued From Part One Monitoring Adherence to Non-Opioid Treatments Given the links between chronic pain and mental health, a large part of managing chronic…
Amanda Gilpin, a resident of Karratha in Western Australia, noticed that her little daughter occasionally would roll her eyes in response to some of her motherly advice. At times, she…
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…
Chloe Goodman is no stranger to tough situations. As a model and reality TV star from MTV UK's Ex on the Beach, Chloe has certainly found herself in stressful environments. But…
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