The US FDA has approved the first drug to treat neurotrophic keratitis, a degenerative condition that affects the eyes. Approval of the drug, called Oxervate (cenegermin), is granted to…
According to a story from FiercePharma, the US Food and Drug Administration (FDA) has recently ordered a total of 13 different diabetes drugs to begin carrying warning labels about the…
According to a story from prnewswire.com, the drug Cablivi, also known as caplacizumab, has been granted marketing authorization by the European Commission. Cablivi is meant to treat acquired thrombotic thrombocytopenic…
The rare disease advocacy community is mourning the loss of a wonderful voice, Claire Wineland, who passed away Monday after her battle with Cystic Fibrosis. Claire suffered a stroke on Aug…
According to a story from Newswise, the results of a phase 3 clinical trial suggest that the medication tafamidis could substantially reduce the risk of hospitalization or death for patients…
Happy Last Day of August! As we get ready for the autumn, we want to highlight four articles. We have an article about the Zebra Ball fundraising for EDS advocacy,…
Happy Last Day of August! As we get ready for the autumn, we want to highlight four articles. We have an article about the Zebra Ball fundraising for EDS advocacy,…
According to a story from BioSpace, the gene therapy company Reflection Biotechnologies Ltd. recently announced that the US Food and Drug Administration (FDA) has given Orphan Drug designation to its…
According to a story from globenewswire.com, the pharmaceutical company Novartis recently announced that its CAR-T cell therapy drug Kymriah recently gained approval from the European Commission. The drug was approved…
According to a story from Akcea Therapeutics, the company recently received a reply from the US Food and Drug Administration (FDA) in regards to their New Drug Application for WAYLIVRA…
For comedy and Saturday Night Live fans, Pete Davidson needs no introduction. For those out of the loop, you might now be hearing more of this young comedian who is…
According to a story from BioSpace, the biopharmaceutical company AbbVie recently announced that the US Food and Drug Administration (FDA) has approved a new combination treatment for Waldenstrom's macroglobulinemia (WM).…
According to a story from globnewswire.com, the gene therapy company Adverum Biotechnologies recently announced that the US Food and Drug Administration (FDA) has granted them Orphan Drug designation for their…
According to Digital Health Age, a wearable eye technology may be revolutionary in the lives of those who have Locked-In Syndrome, which is often associated with ALS and other motor…
According to a story from BioSpace, the US Food and Drug Administration (FDA) recently approved the drug Oxervate (cenegermin). This treatment is the first FDA approved therapy for the rare…
Happy Back 2 School Time! As the week comes to a close, we want to spotlight four articles. We have an honest piece from PW contributors Denise Crompton and Tom…
According to a story from BioSpace, Martin Pharmaceuticals' Livantra appears to be on something of a roll. Earlier in the year, the drug received Orphan Drug designation for the treating…
According to a story from Financial Buzz, the drug development company Eisai and Merck recently announced that their cancer drug lenvatinib, marketed as LENVIMA,® has gained marketing approval from the…
According to a story from hemophilia.org, the drug developer uniQure recently announced that the first patient had been dosed in the company's Phase IIb clinical trial. This trial is testing…
According to Science Blog, a therapeutics company is on its way to creating an implantable, insulin-producing device that may give injections a run for their money. Therapies similar to…
Several organisations are collaborating to develop a gene therapy that targets cystic fibrosis. Boehringer Ingelheim, Imperial Innovations, Oxford BioMedica, and the UK Cystic Fibrosis Gene Therapy Consortium will work…
The Alagille Syndrome Alliance has launched its first ever awareness ribbon in support of the Alagille syndrome (ALGS) community! The multi-colored awareness ribbon is meant to represent the complex and uniquely…
According to a story from EurakAlert!, a group of researchers from the University of Pennsylvania School of Medicine have achieved a major breakthrough for idiopathic pulmonary fibrosis research that will…
According to a story from Morningstar, the company Alexion Pharmaceuticals recently announced that the U.S. Food and Drug Administration (FDA) has agreed to review their Biologics Licensing Application for their…
According to the Santa Cruz Sentinel, Josalyn Catarino is an 11-year-old who loves Disneyland, TV romance shows, the ocean, and getting her fingernails painted. This, of course, sounds like any…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
