According to a story from MedCity News, the drug company Eli Lilly was anticipating the approval and launch of its drug mirikizumab as a treatment for ulcerative colitis. However, the…
The passage of the Orphan Drug Act was an important step in creating an environment that supports rare disease research and drug development. This Act created Orphan Drug designation,…
In February 2023, I spoke with Dr. Allen Davidoff of XORTX Therapeutics Inc. (“XORTX”) about the company’s commitment to developing therapies for people living with progressive kidney disease. We…
According to a story from BioPharma Dive, the drug company AbbVie has recently announced its decision to withdraw two different approvals for its cancer therapy Imbruvica. The approvals were withdrawn…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
STAT NEWS recently published an op-ed with a provocative question: How do we tell the difference between legitimate mental health tech and snake oil? Looking back to the early…
On March 22, 2023, via PRNewswire, the FDA announced that its CNS Drugs Advisory Committee voted unanimously in favor of a potential accelerated approval of tofersen to treat people with…
On February 1, 2023, FDA issued a Draft Guidance Document, called “Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products” (Docket ID: FDA-2022-D-2983). The Draft Guidance considers…
Have you ever heard of the Orphan Drug Act? This Act helped stimulate and advance the development of, and research into, therapeutics for individuals living with rare or underserved conditions. In…
Theravance Biopharma’s late-stage failure suddenly became a jump-off point for treating multiple system atrophy (MSA) according to a recently published article in Neurology Live. After the failure of two…
Revolo Biotherapeutics was founded with one specific goal in mind: to benefit people living with autoimmune and allergic diseases by creating novel, life-changing therapeutics that induce long-term remission. They…
Discovering a treatment for a rare disease is challenging but it can also be rewarding. This is especially true when treating an extremely rare disease such as perivascular epithelioid…
Ayla Bashir and her parents, Sobia Qureshi and Zahid Bashir, have participated in a medical first with Ayla surviving Pompe disease. The same disease caused the death of her…
The National Institutes of Health (NIH) held its in-person Rare Disease Day at NIH convergence on February 28, 2023. This was the first time in a while that the event…
STATHMIN-2 (STMN2) is a protein that has been proven to play a role in axonal development, repair, and stability. This protein is highly expressed in human motor neurons; research…
Currently, the standards-of-care for narcolepsy include lifestyle changes involving diet and exercise, behavioral therapy, nap therapy, and certain medications designed to combat excessive daytime sleepiness. However, a majority of…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
According to reporting for Cure Today, the FDA recently granted Accelerated Approval to a combination treatment of Padcev (enfortumab vedotin-ejfv) and Keytruda (pembrolizumab) for advanced or metastatic urothelial carcinoma.…
In the United States, Orphan Drug designation is granted by the U.S. Food and Drug Administration (FDA) to drugs or biologics that are being developed for rare conditions. Rare…
The Ovarian Cancer Research Alliance stated in new guidance that evidence indicates the majority of ovarian cancers begin in fallopian tubes. The president of the alliance, Audra Moran, was…
Statins are a type of drug that can help you to lower your blood cholesterol levels. These drugs work by inhibiting HMG CoA reductase, an enzyme that the body…
Approved for use in 2017, emicizumab—marketed under the brand name Hemlibra—has shown immense benefits in the treatment of hemophilia A. But researchers wanted to better understand how emicizumab, used…
The complexity of rare diseases makes communication between adults involved with the care of these children a vital tool. Communication and mutual trust are of the utmost importance. About the…
JScreen is a non-profit organization headquartered at the department of Human Genetics at Emory University in Atlanta. JScreen offers genetic testing and educational services, such as genetic counseling. JScreen's mission…
In 2022, the U.S. Food and Drug Administration (FDA) examined the potential of approving palovarotene, a selective RARγ agonist, for the treatment of fibrodysplasia ossificans progressiva (FOP). However, the FDA…
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