In a news release from biopharmaceutical and RNAi therapeutics company Alnylam Pharmaceuticals, Inc. ("Alnylam"), the company shared that positive data was available from the Phase 3 APOLLO-B clinical trial.…
Written by Muriël Marks, Executive Director, World Alliance of Pituitary Organizations History Between 2012-2014, Novartis started to send out personal invitations to acromegaly and Cushing’s patient advocates to represent their…
Targeted Oncology recently published an article describing a group of T cell malignancies associated with blood disorders (hematologic cancers). Currently, a high rate of patient deaths and relapses are occurring…
For most of his life, Jack O’Donnell was as healthy as can be. Even as he headed towards Oklahoma State University, ready to study engineering graphics and design drafting, Jack…
In the U.S., the FDA grants Orphan Drug designation to drugs or biologics intended to treat, diagnose, or prevent rare diseases or conditions. “Rare” refers to any condition affecting fewer…
Inside Precision Medicine recently featured an article describing a study that was published in the European journal Diabetologia conducted by a team of Chinese and Swedish researchers. The study suggested…
According to a story from Market Watch, the drug companies Merch and Eisai recently announced an update on their phase 3 clinical trial. This clinical trial was evaluating a combination…
Kris and Carla Thompson were so thrilled when they learned that they would be expecting a daughter. When Lucy was first born, Carla describes her as a cheeky, upbeat, and…
On July 27, 2022, the National Organization for Rare Disorders (NORD) hosted an online webinar titled "Drug Development for Rare Diseases: A Community Conversation." This program was designed to help…
According to a news release from biotechnology company Kira Pharmaceuticals, its therapy KP104 earned Orphan Drug designation in the United States. KP104 is in development as a therapeutic option…
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…
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In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare or life-threatening conditions. A “rare” condition is defined as…
Results of a recent study published in Medscape Medical News assessed the effect of treatment with radiotherapy on patients who have prostate cancer that is localized. Statistics show that…
An article published by Ophthalmology Times Europe shared that the first patient had been dosed in the Phase 2 STARLIGHT clinical trial evaluating MCO-010 as a potential therapeutic option…
When Mercedes Young found out that she was pregnant with her son Milo, she knew that she would do everything possible to protect and support her son. Milo was born…
According to a recent article in the British publication Latest Page News, doctors at the University College in London and Royal Free London believe that a ‘cure’ is possible within…
Pursuing an Alzheimer’s disease diagnosis can not only be costly, but time-consuming. It may take numerous neuropsychological examinations, alongside other testing and analysis, to confirm this diagnosis. The issue is…
August has begun and you know what that means – it is Spinal Muscular Atrophy (SMA) Awareness Month! During this month, many patients, advocacy groups, family members, and others work…
Note: Research Square publishes preliminary, non-peer-reviewed scientific reports. Therefore, while the data below is interesting, it is not academically published and, as stated within the published article on Research Square: "the…
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…
The month of August is recognized as Stevens-Johnson Syndrome Awareness Month. The goal of this month is to elevate awareness about Stevens-Johnson syndrome among the general public and in the…
According to a story from Myasthenia Gravis News, the European Medicines Agency Committee for Medicinal Products for Human Use (CHMP) has recommended ravulizumab (marketed as Ultomiris) to be approved as…
Currently, the standards-of-care for treating medulloblastoma include surgical interventions, chemotherapy, and radiation. Finding new and effective treatment options would help to improve patient outcomes. According to an article in The…
According to a news release from biotechnology company Sound Pharmaceuticals, enrollment has begun in the Phase 3 STOPMD-3 clinical trial. So far, the first patient has been enrolled – with…
Rare Disease Diagnoses Get a Boost from Scientists’ New guidelines Professor Dame Sue Hill, Chief Scientific Officer of NHS England, recently interviewed with MedicalXpress citing the importance of unraveling the mystery…
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