According to a story from sciencex.com, Queen's University Belfast, University College Dublin, and a team of 33 other partners have come together to start the All-Ireland Rare Disease Interdisciplinary Research…
Also known as Lawrence syndrome, acquired generalized lipodystrophy (AGL) is an ultra-rare condition characterized by the destruction or loss of adipose tissue. Given the small population size, AGL is…
According to a story from koin.com, graft-versus-host disease is a condition that can affect recipients of stem cell or organ transplants. Dr. Corey Cutler, Medical Director, Stem Cell Transplant Program,…
For the last eight years, the National Organization for Rare Disorders (NORD) has developed a State Report Card; the goal of this project is to evaluate the effectiveness of…
Reducing the spread of viral illnesses is of crucial importance to countries around the world. One such viral illness is Zika virus, a mosquito-borne illness that can be extremely…
According to a story on pharmaphorum.com, the UK's National Institute for Health and Care Excellence (NICE) has given a positive opinion for the drug vutrisiran (marketed as Amvuttra) as a…
The Orphan Drug Act was passed with a goal in mind: to incentivize the development of therapeutics for orphan drugs. These drugs are used for patients with rare conditions,…
Shifts in treatment paradigms can improve the lives of countless patients and contribute to better overall outcomes. In a news release from February 23, 2023, Sanofi and international biopharmaceutical…
According to a story from World Pharma Today, Andelyn Biosciences, Inc., which is a gene therapy Contract Development and Manufacturing Organization (CDMO), has recently struck a partnership with Odylia Therapeutics,…
According to a story from VICE News, Congressman George Santos was recently elected to Congress in the 2022 mid-term elections. Since then, his past has been placed under scrutiny, and…
Six years ago, the United MSD Foundation began its annual Zebra Run with a goal of raising multiple sulfatase deficiency (MSD) awareness, as well as garnering funds to advance…
Until recently, the only therapeutic options for people with Friedreich ataxia were symptomatic and focused on symptom management: mobility aids, surgery, psychotherapy, speech therapy, heart medications, hearing and vision…
According to a story from Globe Newswire, the gene therapy company uniQure recently announced that it has received conditional marketing authorization for its gene therapy etranacogene dezaparvovec (marketed as HEMGENIX®).…
From February 28 to March 2, rare disease advocates met in Washington, DC for the 12th Annual Rare Disease Week on Capitol Hill. Organized by Rare Disease Legislative Advocates (RDLA),…
February 28th is recognized as Rare Disease Day around the world each year. This day is intended to bring recognition and awareness to the plight of the millions of people…
In March of 2021, twenty-eight-year-old Alix Burnard of Newbury, Berkshire UK had several Covid-19 tests due to a suspected infection. All three tests were negative, so she felt it…
In the United States, Orphan Drug designation is granted to drugs or biologics designed to treat, prevent, or diagnose rare conditions. “Rare” conditions refer to those affecting under 200,000…
Imagine being Rowan Marshall, a seven-year-old first-grade student with a debilitating rare disease. Rowan made it through the new student formalities. He is doing well at school. Now he faces…
Researchers have been grappling for years with the STAT5 protein as a tool to fight cancer. STAT5 is critical because it is partly responsible for the development and progression…
After winning the 1998 100m butterfly in Perth, breaking a world record, winning 54 international medals, and becoming the Swimming Hall of Fame inductee in 2022, Michael Klim appeared…
In June 2022, Andrew Mace received CPR from a neighbor, 21-year-old Alex Duncan, a med student who luckily remembered her instructions while at Cambridge University, UK. Andrew, age 44,…
The failure rate of drug development for Alzheimer’s is 99 percent according to a recent study. Since 1984 scientists have concentrated on developing treatments to prevent the formation of…
Currently, a glioblastoma multiforme (also known simply as glioblastoma or GBM) diagnosis comes with a poor prognosis. There is a 40% survival rate after one year which falls to…
In the United States, Orphan Drug designation is granted to drugs or biologics being developed to treat, prevent, or diagnose a rare disease. These are conditions affecting fewer than…
The Foundation for Angelman Syndrome Therapeutics (FAST) recently entered a partnership with Rush University in order to establish a clinical trial and translational research center for rare neurodevelopmental disorders.…
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