According to a story from Business Wire, the pharmaceutical company Takeda recently presented results from its phase 3 clinical trial evaluating HYQVIA (Human Immune Globulin Infusion 10% with Recombinant Human…
NS Pharma, Inc., a subsidiary of Nippon Shinyaku Co., recently announced via news release that its therapy NS-089/NCNP-02 was granted Orphan Drug designation from the U.S. Food and Drug…
Even with available therapies, pancreatic cancer is incredibly tough to treat and comes with a poor prognosis. Only 12% of people diagnosed with pancreatic cancer are still alive after…
According to a story from Greenock Telegraph, Michael Conway of Port Glasgow, UK lives with adrenoleukodystrophy, a rare disease. Recently he received a letter of support from William and Kate,…
Between 2019 and 2020, the number of reported Hansen’s disease (leprosy) cases across the United States fell. However, there have been an uptick of cases since 2020, especially in…
65 roses. This phrase is often used in reference to cystic fibrosis; in fact, the origin of 65 roses is a young boy who couldn’t properly pronounce the name…
According to a story from BioPharma Dive, the drug company BridgeBio has been working on the development of a new treatment for limb-girdle muscular dystrophy, a rare disease. At the…
Each summer advocates for rare diseases take advantage of longer daylight hours and use the opportunity to showcase the hopes, needs, and wants of people with rare diseases such as,…
According to a story from wtae.com, doctors didn't think that 11-year-old Bryson Ackermann would be able to return to playing baseball after he was diagnosed with acute flaccid myelitis, a…
According to a story from Global Genes, the US Food and Drug Administration (FDA) has said that it will not take the pharmaceutical company Biohaven's recent New Drug Application under…
It can be difficult to grasp the scope of a rare disease, its symptoms, and impact—especially if certain individuals don’t fall within the understood or narrowly defined parameters of…
According to a story from Cure Today, the biopharmaceutical company Ambrx Biopharma, Inc., recently announced that the US Food and Drug Administration (FDA) has given Fast Track designation to the…
The success of the CARES 310 Trial Phase III (NCT03764293 ) was reported this week in Biospace by Elevar Therapeutics. The combination of two drugs, rivocera, and camrelizumab proved to…
The long-awaited decision by the European committee CHMP was announced recently by Amylyx Pharmaceutical (the Company) based in Cambridge, Massachusetts just as anticipated. The EMA issued its negative opinion of…
People in China who are living with narcolepsy will soon have access to a new therapeutic option to help them manage cataplexy or excessive daytime sleepiness (EDS) after the…
Patient Worthy partner Chelsea's Hope is a nonprofit organization dedicated to supporting families impacted by the deadly rare illness Lafora disease, as well as amplifying research. Recently, a documentary titled Fighting…
According to a story from Vox, Americans are some of the world's most voracious meat eaters. Per capita, Americans eat an average of 60 lb. of red meat each year,…
According to a story from Market Screener, the drug company AbbVie recently announced that its investigational therapy epcoritamab has received a positive opinion from the European Union's Committee for Medicinal…
According to a story from WGN-TV Chicago, treatment for esophageal cancer can be grueling on patients, even if successful. Surgery is often a critical aspect of the process and can…
Prothena Corporation which is based in Dublin, Ireland recently announced, via Business Wire and Biospace results of the VITAL Phase 3 clinical trial showing a significant survival benefit…
Findings from the Max Delbrück Center and the Charité Universitätsmedizin Berlin have been published in the European Respiratory Journal stating that a triple combination therapy can improve CF symptoms…
If your child was sick, you’d do everything that you could to save them. Hayley Moore is no different. When her daughter Klarissa was born, Moore felt such a…
According to a report in the morningstar.com, the FDA has approved AstraZeneca/Sanofi shot that protects infants and toddlers against RSV. The 2022/2023 winter saw an RSV increase, especially among…
According to a story from European Pharmaceutical Review, the European Commission has recently approved a label extension of ORKAMBI (lumacaftor/ivacaftor), a drug used to treat cystic fibrosis. Under the extension,…
According to a July 11 article from Maaisha Osman, commercial-stage biotechnology company ADC Therapeutics has voluntarily halted enrollment for the Phase 2 LOTIS-9 study evaluating Zynlonta (loncastuximab tesirine-lpyl) and…
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