Mazidul Islam told the Daily Star that he had to watch his 14-year-old son Farid die and could do nothing to save him. Among other things, he regrets the fact…
An antidepressant drug that was approved by the FDA in 1959 is being put to new and promising use to fight hard-to-treat breast cancers. The antidepressant, imipramine, was approved to…
It’s no secret that the COVID-19 pandemic significantly changed the way we interact with the world around us: in our social lives, the way we work, the way we communicate,…
Michael Myers, M.D., CEO of the Quoin pharmaceutical company describes Netherton Syndrome as a devastating skin disorder that at times may be fatal. In a recent article published in BioSpace,…
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…
Cognitive deficits have been recognized as symptoms of advanced liver disease for many years. As reported in Bioengineer, the American Journal of Pathology recently published a study providing insights…
The 64th ASH Annual Meeting held on December 10, 2022, focused on various approaches for hematologic diseases with the goal of improving quality care and outcomes. Dr. Stephanie Lee, of…
Jenny Decker of Fenton, MO. interviewed with KMOV4 explaining that she was unable to walk when she was three years old due to a rare disease called Charcot-Marie-Tooth disease. Doctors…
Sangamo Therapeutics, with its primary focus on genomic medicine, recently issued a news release announcing data from its phase 1/2 STARR trial. The data evaluated isaralgagene civaparvovec, or (ST-920) as…
In 2021, the U.S. Food and Drug Administration (FDA) approved zanubrutinib (Brukinsa) for adult patients with relapsed or refractory (R/R) marginal zone lymphoma. At the 64th Annual American Society for…
In the United States, Fast Track is the process designed to facilitate the development, and speed up the review, of therapies designed to fill an unmet need or treat…
According to a story from myscience.org, neurodegenerative diseases, such as amyotrophic lateral sclerosis, are diseases that progressively worsen over time. However, progression in this disease is not consistent across all…
According to a story from hopkinsmedicine.org, a study evaluating data from 106 patients living with wet age-related macular degeneration found a significant disparity in outcomes when comparing patients that received…
Rare disease stakeholders are called on to participate in the first Rare Disease Therapeutic Alliance of 2023 featuring an agenda presented by Rare Advocacy Movement experts. The agenda will…
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…
423 words 10% matched vs 524 words 5% matched The Rare Diseases International Organization reports that the groundbreaking UN Resolution on Persons Living With a Rare Disease (PLWRD), effective…
According to a story published on Charcot-Marie-Tooth News, a recent real-world study highlighted the impacts and symptoms of Charcot-Marie-Tooth (CMT) disease type 1A. The most common form of the illness,…
Before you read on, make sure to check out: Part 1: Green Bay Packers' Shemar Jean-Charles and Los Angeles Chargers' Storm Norton talk about the importance of Alzheimer's disease awareness. …
One year ago, in December 2021, clinical studies of lovo-cel for patients who were eighteen or younger were put on partial hold by the FDA. At the time, bluebird…
Over thirteen years ago, Josh McGuigan met his wife Stephanie – and the rest was history. In the time since, the couple has gotten married and created a beautiful life…
Initially, when doctors in Malaysia met with the 21-year-old patient, they suspected that he had mandibuloacral dysplasia. The man, 21 years old, was short in stature (3.8 ft tall)…
In early January 2023, Healio reported that IOA-244, a treatment designed for uveal melanoma, was granted Orphan Drug designation by the U.S. Food and Drug Administration (FDA). Orphan Drug…
Orphan Drug designation is granted by the FDA to drugs or biologics intended to treat, diagnose, or prevent a rare disease or condition. “Rare,” in the United States, is…
Before you read on, make sure to check out: Part 1 of our article: Green Bay Packers' Shemar Jean-Charles and Los Angeles Chargers' Storm Norton discuss why they raise Alzheimer's disease…
Simeon Fryer was elected captain of the Texas A&M University Corpus Christi (TAMUCC) Islanders men’s basketball team for the 2022-23 season – and it’s a job that he takes very…
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