A recent study has uncovered that a CAR-T therapy called Kymriah could lead to improved results for follicular lymphoma patients. Specifically, it was found to be both effective and safe…
In a recent news release, commercial-stage biopharmaceutical company HUTCHMED shared that its investigational therapy HMPL-523 had received Breakthrough Therapy designation from the National Medical Products Administration (NMPA) in China. HMPL-523…
Prior to February 2022, there were no approved disease-modifying therapies for those with pyruvate kinase (PK) deficiency. However, this has recently changed! According to a news release from biopharmaceutical company…
Rare Disease Week 2022 Rare Disease Week is the week that leads up to Rare Disease Day, which is recognized on the last day of February. This year, we are…
On February 16, 2022, biotechnology company Hillstream BioPharma Inc. (“Hillstream”) shared that its drug candidate HSB-1216 received Orphan Drug designation from the U.S. Food and Drug Administration. This particular designation…
On February 15, 2022, biotechnology company Zealand Pharma A/S (“Zealand”) announced that its Phase 3 1703 clinical trial evaluating dasiglucagon for congenital hyperinsulinism (CHI) had reached full enrollment. Within this…
Researchers are consistently looking for ways to deepen their understanding of various conditions, which not only helps with drug development but with patient outcomes. According to Medical XPress, a research…
According to a recent article, a cyclist who was closely affected by alpha-1 antitrypsin deficiency (A1AD), is biking across Australia with his good friend to raise awareness and money for…
Prozac (fluoxetine) is used to treat a variety of mental health conditions including major depressive disorder (MDD), obsessive-compulsive disorder (OCD), bulimia, and premenstrual dysphoric disorder (PMDD), among others. You may…
An article recently came out encouraging people who are experiencing a blocked nose to not just dismiss it as a common cold, as it could be nasopharyngeal cancer (NPC). Nasopharyngeal…
According to a recent article, after the results of a clinical trial, researchers have proven that CAR-T cells can cure patients that are diagnosed with leukemia. Leukemia Leukemia is a…
Rare Disease Week 2022 Rare Disease Week is the week that leads up to Rare Disease Day, which is recognized on the last day of February. This year, we are…
In Part 1 of our interview with Lorrie and Stewart Altman, we discussed the journey to the Late-onset Tay-Sachs disease (LOTS) diagnosis, how Lorrie and Stewart's outlook shaped their lives,…
Alnylam Pharmaceuticals has just announced that they will be starting a Phase 2 trial for their RNAi therapeutic called Lumasiran. This trial will investigate both the safety as well as…
According to a recent article, researchers in Europe have created a fluorescent biosensor to look at the signaling molecule orexin in mice brains to help understand narcolepsy. Narcolepsy Narcolepsy and…
In a news release from early February 2022, clinical-stage biotechnology company Eureka Therapeutics, Inc. (“Eureka”) shared that two of its therapies – ET140203 and ECT204 – received Orphan Drug designation…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
In late January 2022, biopharmaceutical company BridgeBio Pharma, Inc. ("BridgeBio") shared via news release that the first patient had been dosed in the Phase 1/2 ADventure clinical trial. Within this…
The FDA has just announced that they have granted Orphan Drug Designation to an investigative treatment called eplontersen. This therapy is being studied as a treatment option for transthyretin (ATTR)…
Rare Disease Week 2022 Rare Disease Week is the week that leads up to Rare Disease Day, which is recognized on the last day of February. This year, we are…
48 years of marriage, 2 children, 2 nephews, 7 grandchildren, and 3 great-nieces. It is clear that Lorrie and Stewart Altman, over their time together, have created a beautiful life.…
Stoke Therapeutics and Acadia Pharmaceuticals made a joint announcement this week through Business Wire of their collaboration in the development of RNA-based medicines to potentially treat rare neurodevelopmental disorders of…
CURE® recently conducted an interview with von Hippel-Lindau (VHL) patient Joseph Heisler and his mother, Marirene. Joseph’s father was diagnosed thirty years ago with the genetic (inherited) disease. Shortly thereafter…
According to a relatively recent news release from biopharmaceutical company Maze Therapeutics, the company has begun dosing healthy volunteers in a Phase 1 clinical trial. During the trial, researchers are…
Dr. Douglas Marchuk, professor of genetics at Duke, has spent years studying the genetics of cardiovascular disease. According to a recent article in the Atlantic, his research at the University…
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