According to a story from NRI Pulse, Kiran and Anju have been forced to watch over the past year as their daughter Aadya's physical and mental condition declined. After serious…
According to a story from BioSpace, the biotechnology company Partner Therapeutics, Inc., recently announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation to the company's…
According to a story from Hematology Times, an experimental CAR-T cell therapy called P-BCMA-101 was recently granted regenerative medicine advanced therapy (RMAT) designation from the US Food and Drug Administration.…
According to a story from Futurity, a recent study has identified a protein that plays a central role in the onset of a rare cancer called subcutaneous panniculitis T lymphoma…
In the movie "Dallas Buyers Club" the main character, Ron Woodroof, contracts HIV/AIDs and starts getting drugs from an organization that imports medicine from Mexico. Similarly, many London patients are…
In a recent news article, the Northwestern Medicine Feinberg School of Medicine has shared details about an experimental device that may have the potential to dramatically improve the treatment of…
According to a story from prweb.com, the Neuromuscular Disease Foundation (NDF) recently announced that is has earned a $2.5 million challenge grant. This massive grant will go towards funding the…
An investigational new drug for bronchopulmonary dysplasia, called CF-MEV-132, has completed pre-clinical in-vivo testing. According to The Cell Factory, who are developing the drug, the potential treatment is now ready…
According to a story from EurekAlert!, a recent study from the Children's National Health System has found that some cystic fibrosis patients are not getting sufficient doses of antibiotics in…
According to a story from statnews.com, Ella Balasa, who has cystic fibrosis, has been seeking to join a clinical trial in hopes of finding a new treatment that can help…
This is part two of Morgan's story. Read the first half here. I was taken off of work and school for three months to monitor my condition. I had to drop…
According to a story from Vietnam News, mother Huyen Trang has seen two of her children face death's door within mere months of being born. Her first child died at…
BioMarin has just released an update on their achondroplasia and hemophilia clinical trials. Exciting things are in the works! Achondroplasia Update Achondroplasia causes short stature. A phase 3 trial working to…
In 1993, researchers found one of the causes of familial amyotrophic lateral sclerosis (ALS). ALS is a neurodegenerative disorder which affects the motor neurons. Motor neurons are responsible for transmitting…
A new published research study shows that women who breastfeed at least one child for six months or more are at a lower risk of having nonalcoholic fatty liver…
Happy Friday! Winter is coming and so is FDA news! Take a moment to check out some recent FDA announcements. FDA Approves Triplet Immunotherapy Combination for Multiple Myeloma Big news…
This is part one of Morgan's journey with POTS. Check out the second half of her story here. Like most stories, mine begins with someone else’s. I was in sixth…
According to a press release by the World Health Organization (WHO), the latest statistics from The International Agency for Research on Cancer (IARC) provide a new picture of the global…
According to a story from the Ottawa Citizen, Dianne Sauvé, who is 60 years old, learned six years ago that she has the rare condition acromegaly. Since she learned that…
In a recent news release, Moorfields Eye Hospital Dubai outlines how they effectively treated a young girl for Stevens-Johnson syndrome, a rare and chronic disease that affects the eyes. About…
According to a story from Trend News World, mother Orvelina Lizeth Melendez is heading north as part of the migrant caravan that has been making headlines as it makes its way…
Are you interested in learning about the latest trends and drug development in the rare disease field? Then you should check out a special Journalist Team Presentation from Global Data…
According to a story from telegram.com, a group of researchers from the University of Massachusetts medical school recently announced that they may have reached a major treatment breakthrough for genetically…
Neurofibromatosis is an extremely rare condition. Worldwide it affects just one in four thousand people. Gail Appelgren and Tim Golumbia are two of the individuals living with the illness and…
Hello. My name is Stephanie Lewis. I am 48 years old and have been ill over half my life. I have been diagnosed with Asthma, CFS, IBS/C, Interstitial cystitis, dysautonomia,…
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