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,…
Epidiolex was the first cannabis-based drug approved in the United States. It was first recommended for FDA approval by an advisory committee in April of 2018. In June of this…
Society teaches us to make comparisons all of the time. When you chose to participate in a competitive game, and you want there to be a winner and a loser,…
According to a story from Fierce Healthcare, the decision by the Trump administration to curtail planned efforts to update standards of care for people with disabilities means that this community…
According to a story from Medical Xpress, a team of researchers from the American Association of Ophthalmology recently released a study that highlights the dangers of unapproved stem cell therapy.…
While I write about rare and neglected disorders all the time, very few people in my life actually know about my own experience. I have hypermobile Ehlers-Danlos syndrome (EDS), a…
In a recently shared article, the University of Southern California has announced that the United States Food and Drug Administration has approved a high-tech brain scanner at the Keck School…
According to a recent article by UMass Medical School, promising new research from scientists at UMass supports the potential of gene therapy as a treatment for amyotrophic lateral sclerosis. The…
According to a story from BioPortfolio, the biopharmaceutical company SpringWorks Therapeutics recently announced that the US Food and Drug Administration (FDA) has given the company's experimental product PD-0325901 Orphan Drug…
According to a story from investors.amgen.com, the drug developer Amgen recently announced its intent to present all new clinical trial data which will feature its latest work in testing a…
My name is Jasmine Taylor and I am 12 years old. October is one of my favorite months (July is the other one- it’s my birthday month!)… Let me tell…
According to a story from Business Wire, the drug developer Bioverativ, Inc., recently announced its intention to present data related to its latest developments in blood disorder therapies. This data…
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