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…
At 41 years old, Teddi Mellencamp has done many great things in her life. The daughter of singer-songwriter John Mellencamp, Teddi is a successful podcast host and a reality television…
Written by: Kala McWain There is no amount of time that can fully prepare you for the hard work and sacrifices it takes to be a good parent. These challenges…
Before you read on, make sure to check out Part 1 of our article. In Part 1, Green Bay Packers Cornerback Shemar Jean-Charles discusses why he supports the Alzheimer's Association and how…
Currently, C5 inhibitors such as eculizumab and ravulizumab are considered the standard-of-care for people living with paroxysmal nocturnal hemoglobinuria (PNH). Unfortunately, not all patients respond well to these therapies.…
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…
Throughout his life, Gavin Miller has overcome many struggles. As a young child, Gavin always loved hockey. But when he was in a hockey program at age four, he…
While alveolar soft part sarcoma (ASPS) is considered to be relatively slow-growing, it is necessary to carefully manage this rare soft tissue sarcoma to avoid metastasizing. Surgery is one…
The 65th American Society of Hematology (ASH) Annual Meeting took place from December 10-13, 2022. During the meeting, stakeholders in the hematology field came together to discuss trends, research,…
From September to January each year, our home transforms; each Sunday, my boyfriend and I meticulously set up our living room, make snacks, and spend the day watching football on…
Shelby Harris is no stranger to facing challenges; throughout his football career, Harris has remained as a strong and steady defensive lineman with a career 22.5 sacks as a pass…
Continued From Part One Multiple myeloma is caused by the buildup of abnormal white blood cells that form tumors found primarily in bones. Available treatments work for short periods…
Globe Newswire recently published an article announcing positive results from Amolyt Pharma’s Phase IIa proof of concept clinical trial of AZP-3601. Amolyt Pharma, Cambridge, Massachusetts, and Lyon, France, is a…
Before you read on, don't forget to check out Part 1 of our interview. In Part 1, Alex discusses the multi-year diagnostic odyssey to discover that Raymond has VAMP2. Today, we talk…
Before you read on, don't forget to check out Part 1 of the story. In Part 1, Whitney talks about what CDKL5 deficiency disorder (CDD) is and the diagnostic journey for her…
On December 23, 2022, President Joe Biden officially signed the Consolidated Appropriations Act, 2023. This bill sets in stone the federal government's budget for the year, so naturally it includes…
Part 1 Newswise recently published exciting news of a therapy that causes the immune system to destroy cancer cells in the bone marrow. Researchers at Icahn School’s Tisch Cancer Center…
In the United States, rare diseases are defined as those affecting fewer than 200,000 people. In the cases of ultra-rare conditions, there is often even less research, less resources, and…
Determining the safest, best, and most effective therapeutic options for patients is crucial to improving outcomes and ensuring optimal health. As shared in a story from MD Edge, researchers…
As our understanding of genetics continues to grow, new gene variants are being identified and linked to the development of various diseases. According to Medical XPress, a research team from…
Unfortunately, the prognosis associated with glioblastoma is poor; some studies suggest that survival rates are 40% within one year of diagnosis and 17% within two years, with less than 7%…
Waldenstrom macroglobulinemia (WM) is defined as a B-cell lymphoplasmacytic lymphoma. Data from patients in Latin America on WM are scarce. Researchers are hoping to fill the void by analyzing…
Before you read on, don't forget to check out Part 1 of our interview. In Part 1, Wendy discusses what hypertrophic cardiomyopathy (HCM) is, her rare variant, the diagnostic journey, and her…
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