Singer Celine Dion recently acknowledged that she has an extremely rare neurological disorder called stiff person syndrome (SPS). According to a recent article in The Metro, the disease occurs…
According to a story from The Post and Courier, Chantel Bass and her family have been living in the shadow of a rare immune illness called chronic granulomatous disease (CGD).…
New Delhi: Recent headlines in the Hindustan Times highlighted a letter written by the leader of the Bharatiya Janata Party, Varnum Gandhi, to Health Minister Mansukh Mandaviya urging him…
Ashley Colburn began losing her vision in childhood and became completely blind at fourteen years old. You see, Ashley has a rare inherited disorder called von Hippel-Landau syndrome (VHL),…
Sabiha Aoudia and Atmane Abbas were visiting the United States from Algeria when, suddenly, Aoudia began experiencing some pregnancy complications. She underwent an emergency C-section and Alice, the couple's…
Before you read on, don't forget to check out Part 1 of our interview. In Part 1, Dr. Raghavan discussed his background, explained what lupus is, and introduced some of the findings…
After Tabitha Wright heard the news from the doctor, her heart dropped. Her daughter Aspen had just been diagnosed with spinal muscular atrophy (SMA) type 1, a rare genetic…
On January 6, 2023, the FDA granted conditional approval to a new Alzheimer’s drug, lecanemab, that will be sold as Leqembi. Vox carried the original story and included an…
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,…
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