According to a story from Raconteur, finding sufficient funding for the development of new therapies for rare diseases has always been a major challenge. Rare diseases are defined in the…
According to a story from tradeshownews.vporoom.com, the biopharmaceutical company Viking Therapeutics, Inc., has recently released the results from a Phase 2 clinical trial. This trial is testing the company's investigational therapeutic…
According to a story from Benzinga, the drug developers AstraZeneca and Merck have recently announced that the US Food and Drug Administration (FDA) has decided to accept a supplemental New…
According to a story from nature.com, Amber Sapp's twelve year old son Garrett had been participating in a clinical trial that was testing a new potential treatment for his Duchenne…
According to a story from UCI News, University of California Irvine scientists have developed an innovative immunotherapy that could screen patients in order to analyze the composition of their tumors…
According to a story from BioSpace, the gene therapy company Rocket Pharma recently announced that its investigational product candidate RP-L102 recently saw its Investigational New Drug (IND) application gain approval…
According to a story from Market Screener, the genetic medicines company Homology Medicines, Inc., recently announced the presentation of data which revealed that the company's proprietary virus vectors, which are…
According to a story from EurekAlert!, researchers have been scrambling to find a useful treatment for acute flaccid myelitis, a rare disease that has been appearing in unusual numbers in…
According to a story from Scleroderma News, a recent study found that men were more likely to have severe scleroderma early in their diagnosis in comparison to women. This is…
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…
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…
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…
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…
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…
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…
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.…
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…
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