An announcement from the drug company Genentech highlights the results of a recent study which demonstrated that the drug faricimab-svoa (marketed at Vabysmo) was able to dry retinal fluid more…
Ventilatory assistance, NG tubes, tracheostomy: all of these may be used to care for infants born with bronchopulmonary dysplasia (BPD), a chronic lung disease that most commonly affects preterm…
The biotechnology company Anixa Biosciences, Inc. together with the Moffitt Cancer Center, issued a press release on May 22nd stating that the second patient had begun treatment as part…
For patients who were given a diagnosis of advanced, inoperable (unresectable) biliary tract cancer, an article appeared in Healio with some encouraging news. Gemcitabine and cisplatin drugs have…
Omisirge (omidubicel) is the first allogeneic (from a donor) product for SCT that has been the recipient of FDA approval. The FDA's nod moves Omisirge into the realm of…
Republished with permission from Personalize My Medicine, a Patient Worthy partner organization. Written by Eleanor Doherty Glossary Antibody, Antidepressant, Antigen, Apoptosis, Astrocyte, CAR-T Cell Therapy, Atypia, Clinical Trial, CNS, Dendritic Cell Vaccine, Epidermal Growth Factor, Ex-Vivo, Glioblastoma Multiforme, Glioma, Immunotherapy, Metastasis, Mitotic Activity, Monoclonal Antibody, Necrosis, Neuron, Neurotransmitter, Oncology, Oncolytic Virotherapy, Prognosis, Radiotherapy, Stem Cell, Surgical…
Hyperphagia, or excessive and unrelenting hunger, is a key symptom associated with Bardet-Bield syndrome. This excessive hunger often begins around or before 5 years old and can lead to…
On April 25, 2023, myTomorrows, a platform designed to help patients obtain access to experimental medicines, recently launched a new tool called TrialSearch AI, an artificial intelligence-powered resource for physicians…
Our country has an issue when it comes to screening for diabetic retinopathy. In addition to clinics lacking the necessary imaging programs, the compliance rate for diabetic retinopathy screening…
Written by Katherine Tran Lupus is among the most perplexing autoimmune diseases. It can be debilitating when not regulated, with symptoms ranging from butterfly-shaped rashes across the face to fatigue,…
A Business Wire Press Release on April 26, 2023, announced positive interim clinical data on an investigational RNAi therapeutic. Alnylam Pharmaceuticals and its partner Regeneron Pharmaceuticals heralded interim results…
For patients with refractory or relapsed KMT2A rearrangement or NPM1-mutations, the AUGMENT 101 study of the investigational menin inhibitor revumenib yielded impressive results. According to an article in MedicalXpress,…
Clinical trials are often mentioned as an afterthought when discussing the length of time it takes for drugs to be approved by the FDA. On the contrary, clinical trials are…
Prasanna Shirol, Executive Director of the Organization for Rare Diseases India (ORDI), expresses his opinion to the Times of India about the urgency to address the issue of rare…
The FDA estimates that there are about five hundred people in the USA with the SOD1 mutation out of a total of approximately thirty thousand ALS patients. According to a…
KMT2A rearrangements—a DNA fault—lead to treatment-resistant acute lymphoblastic leukemia (ALL) in infants. While around 50% of children with KMT2A rearrangements respond well to intensive chemotherapy, the remaining 50% do…
According to a story from Healio, a recent study found that patients with metastatic pancreatic cancer (more specifically, pancreatic ductal adenocarcinoma, the most common form) that were being treated with…
According to a story from Healio, the US Food and Drug Administration (FDA) has recently updated the indication for the drug teprotumumab (marketed as Tepezza). Approved for the treatment of…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
Currently, there are no cures for amyotrophic lateral sclerosis (ALS). Symptomatic therapy, alongside disease-modifying treatments like Rilutek, Radicava, and Relyvrio, may be used to manage this condition. However, there…
In July 2022, Patient Worthy reported on data from the Phase 3 REAL 4 clinical trial. During the trial, researchers compared the safety, efficacy, and tolerability of Sogroya (somapacitan-beco)…
As bluebird bio ("bluebird") waited on FDA feedback regarding the manufacturing process for lovotibeglogene autotemcel (lovo-cel) genetherapy, the company disclosed that it would most likely miss its Q1 submission…
According to a story from PR Newswire, the biopharmaceutical company Avidity Biosciences, Inc., recently announced that its investigational therapy AOC 1044 has received the US Food and Drug Administration's Fast…
Treatment advances are occurring daily in the field of rare disease. As researchers hone their strategies and tools, new technologies are emerging with the potential to significantly impact patients…
In a news release from mid-April 2023, clinical-stage biotechnology company Abivax SA shared that 2-year results were available from a Phase 2b open-label maintenance study evaluating obefazimod for individuals…
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