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…
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…
Soleo Health has just announced that they have been provided access to lumasiran by Alnylam Pharmaceuticals. This access will allow the company to administer the treatment to patients diagnosed with…
According to a recent article, a new study shows that by increasing your alcohol intake, you may actually be increasing your risk of head and neck cancer. Head and Neck…
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…
In a first-in-human, dose-escalation Phase 1 clinical trial, researchers are evaluating cosibelimab (CK-301) as a potential therapeutic option for those with metastatic cutaneous squamous cell carcinoma (cSCC). So far, shares Cancer…
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)…
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