Professors Susan Treves and Francesco Zorzato, Basel University researchers, have recently presented the first potential approach to congenital muscle diseases. Their report was published in a recent article by Science…
Recent research has given us a better understanding of acromegaly, a rare hormonal disorder. Conducted by Maria Fleseriu, MD, FACE, and colleagues at the Pituitary Center at Oregon Health &…
Pancreatic cancer is typically a difficult cancer to treat, leading to poor outcomes and high mortality rates. One of the reasons for this is the difficulty of diagnosis; it often…
According to a story in the WSJ, the FDA has approved a new therapy for multiple myeloma patients. The therapy is called Carvykti and it is now available as a…
According to a story from Targeted Oncology, the company Genmab has recently announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation to its investigational therapy…
Written by Sumeet Maniar, CEO, WellBrain Continued From Part One Monitoring Adherence to Non-Opioid Treatments Given the links between chronic pain and mental health, a large part of managing chronic…
Written by Sumeet Maniar, CEO at WellBrain In the U.S., 60 percent of all adults have a chronic disease, and 40 percent have two or more. Chronic diseases, which often…
According to a story from AJMC, the drug developer Incyte has made the decision to withdraw its New Drug Application (NDA) for its investigational therapy parsaclisib. This therapy was submitted…
According to a case-based roundtable meeting for Targeted Oncology, Matthew A. Lunning, DO, talked about second-line therapies for diffuse large B-cell lymphoma and the results of a study called L-MIND.…
According to a story from pharmaphorum.com, a therapy being developed for the rare disease activated PI3K delta syndrome (APDS) is on its way to regulators for approval following promising results…
During this year’s commemoration of Rare Diseases, Leonard Saig M.D. a director at Bnei Zion Haifa spoke about Cushing’s syndrome, also called hypercortisolism. Cushing's syndrome is rare and difficult to…
UCB Inc., a biopharmaceutical company, has announced positive results for its RAISE Phase 3 trial (NCT04115293) investigating zilucoplan against placebo. According to a recent article in Biospace, zilucoplan met…
According to a story from Biopharma Dive, the US Food and Drug Administration (FDA) has recently approved a treatment for a rare form of melanoma that affects the eyes, called…
Historically, both osteosarcoma and neuroblastoma have been difficult to treat. Therefore, new treatment options are needed for these cancers, which predominantly affect children and teenagers. According to Medical XPress, a…
In the past, treatment for progressive familial intrahepatic cholestasis (PFIC) was symptomatic or surgical, meaning that there weren't too many treatment options for this patient population. This all changed with…
Tay-Sachs disease has escaped effective treatment since 1883 when an ophthalmologist names Warren Tay found small red spots in the back of the eyes of some infants. According to an…
Research is constantly being done on possible treatment strategies for different forms of cancer; there are many patient populations that face an unmet need. Currently, a team of researchers from…
It's officially Rare Disease Week, which is being celebrated from February 22nd until March 2nd. Every year, we take this week to to recognize, support, raise awareness, and advocate for…
According to a recent article, trametinib has proven to be successful in treating a rare ovarian cancer subtype, which is a major advancement in treating those with this rare ovarian…
Despite undergoing clinical development, many drugs never receive approval from the FDA. Recently, this happened to a treatment for growth hormone deficiency (GHD). The medication, titled somatrogon and owned by…
According to a recent article in SMA News Today, more spinal muscular atrophy (SMA) patients in the United Kingdom (UK) will have access to Zolgensma. This change comes after the…
According to a recent article, the European Commission (EC) has approved Merck’s KEYTRUDA as an adjuvant treatment for type renal cell carcinoma (RCC), a type of kidney cancer. Renal Cell…
According to a recent article, patients with recurring kidney cancer who would most likely need to get their kidney removed can now possibly keep their organ thanks to a new…
According to a study published in PLOS Medicine, only around half of pragmatic or 'real-world' trials collect data as reported by patients or involve patients as partners in research. This…
Recent clinical trials have proven that treatment for relapsed ovarian cancer is not a standard “one size fits all." After a relapse, areas where tumors spread and grow in the…
© 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.
