According to a story from Dr. Hooman Noorchashm, the US Food and Drug Administration (FDA) has a reasonably effective system for regulating and approving new drugs while appropriately evaluating them…
According to an article originally from WCPO Cincinnati, a woman with fibrodysplasia ossificans progressiva is filing a lawsuit against officials of Ohio's Hamilton County. Jessica Scully, 57, alleges that the…
A story from Family Features Editorial Syndicate, published on PR Newswire discussed how getting diagnosed with a chronic, rare disease can be an overwhelming time for a person. While the experience can also give a meaningful…
Happy New Year! For our first Editor's Choice of 2019, we want to highlight four articles from the last couple weeks. First, we have the story of young man who…
Kinetic Kids is a nonprofit which was originally formed to keep children who needed physical therapy active outside of their actual physical therapy sessions by giving them the opportunity to…
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 the Shillong Times, a study published in the New England Journal of Medicine has reported intriguing results when treating sarcoidosis with an already-approved rheumatoid arthritis medication. What is…
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…
What started as a peculiar and insatiable thirst for water led to a rare disease diagnosis for a Denver family. Although they weren't expecting this challenge, they're ready for the…
Nash Jones is 18 months old and he is battling against the clock for a better quality of life. Nash Jones has a very rare genetic disease called Lesch-Nyhan syndrome.…
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Stephanie Hosford was recently interviewed by CURE (Cancer Updates Research and Education) about what she now calls her “three crazy days”. The title of her book is “Bald, Fat and…
The Atlantic featured this incredible story about the possibilities of using genetically altered piglets as avatars for children with genetic diseases. Furthermore, the article delves into the personal story of one…
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…
Dr. Louis J. DeGennaro, who is the CEO and President of the Leukemia & Lymphoma Society, has recently contributed a blog post on the organization's website. In this post, Dr.…
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