Urea Cycle Disorders (UCD) are rare genetic conditions caused by an enzyme deficiency. The urea cycle is responsible for removing excess levels of ammonia from the body. Individuals with UCD…
According to a story from the Irish Examiner, 31 year old Orla Tinsley is celebrating her first year of life following her life saving double lung transplant operation. This lung…
According to a story from BioSpace, the biopharmaceutical company Retrophin, Inc., recently announced that the first patient has been treated in the company's Phase 3 clinical trial testing sparsentan as…
According to a story from CBS News, Garland Benson, age 14, has undertaken a daunting task in order to save the life of his older sister Christiane. She was born…
Lichen sclerosus is a rare, under-researched, and poorly understood condition. The lack of knowledge concerning the disease has led to a lack of awareness, late diagnosis, and a limited amount of…
A Fanconi Anemia (FA) investigative gene therapy now has PRIority MEdicines (PRIME) designation from the EMA. This designation helps to accelerate the development of therapies for patient populations who have…
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For a long time there have been no treatments for Friedreich's ataxia. With no FDA approved therapies, people with this disease have only been receiving symptomatic treatment. Exicure has changed…
Amyotrophic Lateral Sclerosis (ALS) stops communication between the brain and voluntary muscles. Patients slowly lose muscle strength, affecting their ability to walk, talk, and even breathe. The illness eventually results…
People with spinal muscular atrophy and other rare conditions with either few or no approved treatments have recently had their hope of a cure renewed by the concept of gene therapy. Basically,…
According to a story from Science and Technology Research News, a group of researchers from the Cleveland Clinic led by Dr. Aziz Nazha will soon present a new myelodysplastic syndromes…
Cold agglutinin disease (CAD) occurs when the body's antibodies destroy their own red blood cells. It is a form of hemolytic anemia, meaning the body can't produce enough blood cells fast…
What if you could prevent all of the world's most fatal genetic diseases? According to Lee D. Cooper, there's a way- we're just not educating people on how to do…
While rare disease affects people all around us, often the mainstream press . Which is why it's extra important when a news outlet as big as the Los Angeles Times…
According to a story from sectorpublishingintelligence.co.uk, the clinical stage pharmaceutical company Retrotope recently announced that the first patient had been dosed in its Phase 2/3 clinical trial. The trial will…
The views and opinions expressed in this article are those of the authors and do not necessarily reflect the official policy or position of Patient Worthy. According to a story from Vox, a review of over…
According to a story from globenewswire.com, the gene therapy company AVROBIO recently announced that the company's experimental gene therapy drug candidate AVR-RD-01 has earned Orphan Drug designation from the US…
According to a story from gazettelive.co.uk, Jenni Mawson, who is currently 19 years old, has faced a years long struggle with chronic fatigue syndrome, which is also known as myalgic…
According to a story from BioSpace, a report on the results of the Orphan Drug Act of 1983 that was originally commissioned by the National Organization for Rare Disorders (NORD)…
According to a story from CBS News, Roxli Doss, an eleven year old girl, was diagnosed with a rare and dangerous brain tumor called diffuse intrinsic pontine glioma. It is…
Patients, caregivers, and advocates alike all know how devastating and exhausting a rare disease can be, so it's important to find the moments to celebrate when someone overcomes the odds…
Leukodystrophy is the term used for a group of rare, progressive brain disorders which cause the brain's myelin sheath (the nerves protective covering) to either develop improperly or be destroyed…
Methylmalonic Acidemia (MMA) is a liver condition which affects the body's ability to break down food and fatty acids. A dietary imbalance or minor virus for these patients may spark a metabolic…
According to a story from pm360online.com, the biotechnology company Amicus Therapeutics recently announced that the first patient has received treatment in the company's Phase 3 clinical trial testing the company's…
Nontuberculous mycobacterial (NTM) lung infection occurs when someone inhales nontuberculous mycobacteria from the environment. There are various types of this bacteria, giving NTM different forms. But in all types of NTM,…
This story comes to us courtesy of a local Rhode Island TV station's "Small But Strong" news feature, highlighting the strength and tenacity of children. 10-year-old Zoie Mercier and her…
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