According to a story from PR Newswire, Chiesi Global Rare Diseases and the biopharmaceutical company Protalix BioTherapeutics, Inc. have recently announced that they have launched an Expanded Access Program (EAP)…
Disclaimer: This blog post has been republished with permission from the author. To visit the original blog site, click here. Written by Tamara Fowler June 10. My stem cell coordinator…
Healio recently published interviews with members of UCLA’s DRC Health Training and Research Program conducted by Infectious Disease News. Only a few months ago, WHO had announced the end of…
According to a story from the AKU Society, the European Medicines Agency (EMA) has recently announced a positive opinion on extending the label indication for nitisinone (marketed as Orfadin®). The…
aHUS Tracking Atypical hemolytic uremic syndrome (aHUS) is a rare disease that causes blood clots to form within the small blood vessels in the kidneys. These clots lead to organ damage…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
According to an article from BioSpace, the TargetCancer Foundation has began enrolling patients in their TRACK study. This study is intended to provide rare cancer patients with individualized treatment options…
Dr. Jasvinder Singh, a rheumatologist at Alabama’s University in Birmingham is the author of a study that was featured in Arthritis Care and Research. During a recent interview with MedPageToday,…
According to an article from BioSpace, Vivet Therapeutics and Pfizer have entered into a manufacturing agreement to develop Vivet's gene therapy, which treats Wilson disease. The terms of the agreement…
Damian Green from the University of Washington is the Principal Investigator for a new research project in multiple myeloma. This project is thanks to a research agreement between the Fred…
As reported in Image, Sarah of Dublin had her life turned around when at age 10; she became one of the youngest people in Ireland to receive a diagnosis of…
Developmental and epileptic encephalopathies (DEEs), or rare epilepsies, are somewhat of a newer topic. However, researchers are already making strides in creating treatments to address unmet patient needs. On…
Acknowledgment: This story is sponsored by Chiasma, Inc. and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing…
Recently, twenty-two year old Jordan Ray of Wellington, Florida spoke with a reporter from The Palm Beach Post, recounting how her life changed in April 2015 during a softball…
Medical professionals have already discovered a number of genes that play a role in pulmonary arterial hypertension (PAH), but according to LabRoots, they have found a new one. The ABCC8…
Researchers have announced their identification of a specific gene mutation that can lead to atypical hemolytic uremic syndrome (aHUS). This gene is called DGKE and it is not a part of…
Immune-One Therapeutics has just announced that they have dosed their first acute myeloid leukemia (AML) patient in their Phase 1 trial investigating IO-202. This therapy is an immune inhibitory receptor that works…
Just last week, biopharmaceutical company Mustang Bio announced Orphan Drug designation for its therapeutic candidate, MB-207. This unique lentiviral gene therapy is designed for previously treated patients with X-linked…
According to an article from Charcot-Marie-Tooth News, a study was conducted that discovered three new mutations on the GJB1 gene that result in X-linked Charcot-Marie-Tooth disease (CMTX1). The study, which…
Recently, Blueprint Medicines Corporation ("Blueprint") shared that the European Commission (EC) approved marketing authorization of AYVAKYT (avapritinib). The therapy is designed to treat adult patients with PDGFRA D842V mutated gastrointestinal stromal…
Early last week, biopharmaceutical company MediciNova Inc. ("MediciNova") announced promising results for their therapeutic candidate MN-166 (ibudilast). The company, which published in the findings in Cancer Chemotherapy and Pharmacology, explored…
Arrowhead Pharmaceuticals recently announced positive twenty-four-week biopsy results from four patients who participated in the first cohort of the Phase II clinical trial of ARO-AAT. ARO-AAT is Arrowhead’s investigational…
Several decades ago, gene therapy seemed to be on target to treat a disease by simply replacing a defective gene with a healthy one. A recent article republished in APNews…
On September 25, biotechnology company Vertex Pharmaceuticals Incorporated ("Vertex") announced FDA-approval of KALYDECO (ivacaftor) for pediatric patients with cystic fibrosis (CF). Specifically, KALYDECO can treat infants between 4-6 months…
Happy Friday! September is a packed with rare disease awareness, as we've been featuring all month long. We wanted to make sure to draw attention to Dystonia Awareness Month, which…
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