For rare diseases in which no current treatment options are available, such as GM1 gangliosidosis, there is an urgent need to find therapeutics and improve patient outcomes. Gene therapy company…
Rare disease patients often face more obstacles and receive less aid than those impacted by more common conditions. There are many reasons for this inequity, but more research must be…
Have you ever wanted to be part of medical research? Well, now you might have the chance. According to Fox2Now, researchers from Saint Louis University - and, in particular, the…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat patients with rare conditions, defined as affecting fewer than 200,000 Americans. As incentives, drug…
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…
According to a recent press release from Pfizer and biotechnology company Vivet Therapeutics, VTX-801, a gene therapy candidate for patients with Wilson disease, recently received Fast Track designation from the…
Drug development is important in creating new therapeutic options for patients with rare or chronic illnesses. One incentivizing program is the Orphan Drug program. Orphan Drug designation is granted to…
According to a recent press release from biopharmaceutical company NFlection Therapeutics, Inc. (“NFlection”), the FDA granted Orphan Drug designation to the company’s drug candidate NFX-179. Altogether, this treatment is designed…
One key facet of the healthcare system is working to improve patient outcomes and finding new, effective, and innovative lines of treatment. According to Hematology Advisor, a study showed that…
Clinical trials are important tools in determining the safety, efficacy, and tolerability of new therapeutic options. So it is important to learn when patients are dosed, what those dosages are,…
In a recent news release, biopharmaceutical company Retrotope shared that enrollment was complete in its Phase 2 clinical trial. During the trial, researchers will evaluate RT001, a first-in-class oral…
Unfortunately, recurrent ovarian cancer can be difficult to treat and often comes with a poor prognosis. However, according to a news release from biopharmaceutical company IMV Inc. ("IMV"), the company…
Fulcrum Therapeutics recently announced the interim results from their Phase 1 trial of FTX-6058 in a press release. This selective small-molecule inhibitor of EED is in development for the treatment…
For many patients, certain treatments can be expensive, invasive, or difficult to access. But it doesn't have to be this way. According to aHUS News, researchers are running two separate…
Orphan drug designation is granted in the United States to expedite the development and review of drugs and biologics intended to treat patients with rare conditions (over 200,000 Americans). Once…
For years, researchers have evaluated Italfarmaco's givinostat for patients with Duchenne muscular dystrophy (DMD). In fact, a Phase 2/3 clinical trial showed that long-term givinostat treatment helped slow respiratory decline…
HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma patients haven't seen a drug approval for their disease state in a decade. That is until the FDA approved Keytruda in combination with…
Patients with relapsed or refractory mantle cell lymphoma (R/R MCL) may have a new treatment option, based on positive results from a Phase 1/2 trial of Zanubrutinib published in Blood…
On July 30, 2021, biopharmaceutical company Cyclo Therapeutics shared that it had released new and promising data regarding the safety, efficacy, and tolerability of Trappsol® Cyclo™ ("Trappsol Cyclo") for patients with…
Over the last few months, researchers have been enrolling and dosing patients with hereditary angioedema (HAE) from Europe, Israel, and Canada within the Phase 2 RAPIDe-1 clinical trial. According to…
In the past, ibrutinib has been used to treat patients with a variety of conditions, including chronic lymphocytic leukemia (CLL), mantle cell lymphoma (MCL), and chronic graft-versus-host disease (cGVHD). According…
Despite drug developers' best efforts, the path to approval is not always easy. Ask biotechnology company Ardelyx, whose potential treatment option tenapanor was recently rejected by the FDA. According to…
In the United States alone, an estimated 300,000 people have moderate-to-severe chronic idiopathic urticaria (CIU), or chronic hives which are either recurrent or last for over 6 weeks, which…
Previously, biopharmaceutical company XyloCor Therapeutics ("XyloCor") performed the first portion (dose-escalation) of the Phase 1/2 EXACT clinical trial evaluating its investigational treatment XC001 (encoberminogene rezmadenovec) for patients with refractory…
Near the end of June 2021, pharmaceutical company Crinetics Pharmaceuticals, Inc. ("Crinetics") shared via news release that the first patient had been dosed in the Phase 3 PATHFNDR-1 clinical trial.…
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