According to a story from statnews.com, new, groundbreaking forms of treatment, such as gene therapy and CAR-T cell therapy, are giving patients with rare genetic disorders and advanced cancers more…
According to a story from outsourcing-pharma.com, the company Iqvia has just launched its all-new patient portal. However, while many patient portals are intended to allow for patients to monitor their…
The International Waldenstrom's Macroglobulinemia Foundation (IWMF) has recently announced an exciting new partnership that will help facilitate spreading awareness about Waldenstrom's macroglobulinemia (WM), a rare form of blood cancer. This…
According to a story from kfor.com, Governor Kevin Stitt of Oklahoma recently held a signing ceremony for a bill that is being hailed as a rare example of bipartisan collaboration…
Happy Thursday! This week, we're highlighting an article about the neglect of policies meant to protect people with IBD. Next, we have stories on two recent studies affecting MS and…
According to a story from Newswise, Quest Diagnostics, a laboratory that specializes in diagnostic information, has announced plans to participate in a unique, large scale trial called NCI-MATCH, which stands…
Critical Path Institute The Critical Path Institute, or C-Path is a nonprofit organization which was established in 2005. It has a US branch, headquartered in Tucson, Arizona as well as…
Epidermolysis Bullosa Epidermolysis bullosa (EB) is a rare condition affecting approximately 500,000 individuals globally. Patients in the United States and Europe account for 50,000 of these individuals. The hereditary condition is…
According to a story from globenewswire.com, the drug developer ProQR Therapeutics N.V. has recently announced that the US Food and Drug Administration (FDA) has officially cleared the company's Investigational New…
According to a story from BioSpace, the biopharmaceutical company Immunic, Inc. has announced that the first patient has signed up for its proof-of-concept clinical study. This study is intended to…
The headline in a recent Washington Post article declared that the FDA claimed Novartis, through its newly-acquired company AveXis, manipulated data in an application for a gene therapy drug. These…
.jpg){kind=icon}
Trust is vital when it comes to medical care. People put their most important questions and qualms into the hands of doctors and medical staff and expect that they can…
Inflammatory Bowel Disease Inflammatory bowel disease (IBD) is actually a term used to classify a few different conditions. Two of these are Crohn's disease and ulcerative colitis. These are rare…
Juvenile idiopathic arthritis (JIA) is a rare condition affecting children. Like all patients, these children are told what treatment they need and then they are expected to comply. However, researchers in…
According to a story from Financial Buzz, the biopharmaceutical company Anavex Life Sciences Corp. has recently announced that the Independent Data Safety Monitoring Board (DSMB) has completed its review of…
According to a story from Connecticut Children's, the hospital is one of the very first in the country to administer Zolgensma, a recently approved gene therapy for the rare genetic…
According to a story from fda.gov, the US Food and Drug Administration (FDA) has approved the drug pexidartinib (marketed as Turalio) as a treatment for tenosynovial giant cell tumor (TGCT)…
According to a story from First World Pharma, the biopharmaceutical company Mustang Bio, Inc. recently announced that the US Food and Drug Administration (FDA) has approved their investigational new drug…
According to a story from Pharmaceutical Technology, the company Stoke Therapeutics experimental drug candidate, known as STK-001, has earned Orphan Drug designation from the US Food and Drug Administration (FDA).…
RAREsummit19 is a 1 day summit focusing on patient-centricity in rare disease progress. What if we were able to share information with the power to revolutionise the current rare…
Happy Thursday! This week, we're highlighting an story about a woman with Bechet disease who advocates for herself and others. Next, we have an article discussing the difficulty of caring…
Brenda has both psoriatic arthritis and psoriasis. She's lived with the conditions since she was a child, learning to care for herself and deal with the challenges that arose. By…
According to a story from True Viral News, anyone who has tried to get something done through governmental means are probably familiar with getting rebuffed and redirected at every turn.…
According to a story from globenewswire.com, the drug company Amicus Therapeutics recently announced that Argentina's Agency of Medicines, Food, and Medical Devices (ANMAT) has granted approval for the drug migalastat…
According to a story from PR Newswire, the biopharmaceutical company Biohaven Pharmaceutical Holding Company, Ltd. has announced that the first patient has signed up for the company's phase 3 clinical…
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.
