Disruption in clinical trial procedures can be added to the list of changes that have occurred since the start of the pandemic in 2020. The clinical trial sites had always…
An article recently appeared in the AAMC News celebrating the creation of The Children’s National Rare Disease Institute (CNRDI). The article described the long road patients with rare diseases must…
According to YAHOO News, twenty-two-month-old Devdan has a rare disease called type 2 spinal muscular atrophy (SMA2) which affects nerve cells that control muscles. If left untreated progressive muscle weakness…
According to an August 17, 2021 news release from pharmaceutical company Marinus Pharmaceuticals, Inc. (“Marinus”), data is available from a Phase 2 clinical trial evaluating oral ganaxolone for patients…
According to a story from medwirenews.com, a post-hoc analysis from a recent clinical trial has revealed that treatment with filgotinib can result in improvements in structural lesions detected by MRI…
Chimeric antigen receptor (CAR) T-cell therapy has proven to be a successful therapy for a number of conditions; however, we do not see the same success in follicular lymphoma and…
AstraZeneca is ending a phase 3 trial of Ultomiris, which was being evaluated as a treatment for amyotrophic lateral sclerosis (ALS). The pharmaceutical company acquired the C5 inhibitor after buying…
Sometimes, it can be difficult to acquire treatment options for patients with rare diseases. Brand name products may also be expensive and relatively inaccessible, prompting the need for generic…
In 1970, ketamine was used to treat injured soldiers on the tail end of the Vietnam War. The treatment helps relieve pain, but doesn't affect cardiac or respiratory function.…
In a video posted on OncLive, Dr. Anita Kumar, a medical oncologist, discusses different considerations that must come into play when choosing a treatment for people living with relapsed/refractory mantle…
It's no secret that drugs can sometimes be expensive, especially in regards to rare diseases or conditions. But how much would you guess a new drug is priced when…
According to a story on the Renal Fellow Network, rituximab has emerged in recent years as an effective therapy for ANCA-associated vasculitis (AAV). This is a group of autoimmune diseases…
On August 11, 2021, biopharmaceutical company Lantern Pharma, Inc. ("Lantern") shared the news that its small molecule drug candidate, LP-184 (hydroxyureamethylacylfulvene), received Orphan Drug designation from the FDA. This status…
In a video conversation on OncLive, Dr. Daniel Catenacci and Dr. Sam Klempner discuss new biomarkers and therapeutic agents that could have implications for the future treatment of gastric cancer…
According to a story from Pharma Times, a combination treatment consisting of nivolumab (marketed as Opdivo) plus ipilimumab (marketed as Yervoy) will now be available to UK patients living with…
According to a press release from Street Insider, the biopharmaceutical company Fulcrum Therapeutics, Inc., has recently announced positive interim findings from its ongoing phase I clinical trial, which consists of…
According to a story on medicaldialogues.in, a recent study has determined that antifibrotic treatment can reduce the rate of mortality and acute exacerbations in people living with idiopathic pulmonary fibrosis…
A study found on Medscape confirms that the human immune system and the human brain are the most complicated in our biological systems. The two systems are responsible for…
Over ten years ago two Nobel-Prize-winning biologists, Emmanuelle Charpentier and Jennifer Doudna, discovered a technology called CRISPR (clustered regularly interspaced short palindromic repeats). Bacteria contain genetic sequences repeated throughout…
According to a story from Pharma Times, the drug company Bristol Myers Squibb has recently announced that its therapy nivolumab (marketed as Opdivo) has been approved by the European Commission…
According to a story from BioPharma Dive, gene therapy for Fabry disease is still in its early stages of development. However, it is already beginning to show potential as a…
Recently, the FDA approved an investigational new drug (IND) application for '1104, a first-in-class peptide for the treatment of eosinophilic esophagitis (EoE). Now, Revolo Biotherapeutics will move forward to investigate…
In mid-July 2021, biotechnology company Alterity Therapeutics ("Alterity") shared the publication of new data regarding ATH434 for patients with multiple system atrophy (MSA). The data comes from a preclinical…
As reported recently in Medscape, forty-eight patients with myotonic dystrophy type 1 (DM1), were identified by way of the DM-Scope registry, which is the largest collection of data for the DM population. The…
Dr. Javier Munoz, the director of the Mayo Clinic's Lymphoma program in Phoenix, has recently conducted an interview with Kristie Kahl, the Vice President of content for the Cancer Network.…
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