Orphan Drug designation in the United States is granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions, defined as those affecting under 200,000 Americans.…
It is always exciting to see the onset of a new clinical trial designed to research therapies to fill unmet needs for patients. According to a news release from biotechnology…
On October 18, 2021, biopharmaceutical company Paratek Pharmaceuticals, Inc. ("Paratek") shared via news release that the first patient had enrolled in a Phase 2b clinical trial. Within the trial, researchers…
Clinical trials are fantastic ways to develop a deeper understanding of a specific disease, as well as determine the safety, efficacy, and tolerability of potential treatments. Unfortunately, these treatments are…
According to an InBrief PDF from biotechnology and RNA-targeted therapeutics company Ionis Pharmaceuticals ("Ionis"), topline data from a Phase 2 clinical trial highlighted the benefits of cimdelirsen (IONIS-GHR-LRx) for patients…
Canavan disease is an ultra-rare condition that currently has no FDA-approved therapies, meaning that patients face a severe unmet medical need. BridgeBio Pharma aims to change this with their investigational…
In an October 19 news release from biopharmaceutical company Dicerna Pharmaceuticals, Inc. ("Dicerna"), the company shared results from the Phase 1 PHYOX4 clinical trial. During the trial, researchers sought to…
Let us begin this article recently published in MedCity News by starting with the ending: in order to help millions of rare disease patients waiting for rare disease treatments, a…
According to a news release from biopharmaceutical company Clearside Biomedical, Inc. ("Clearside") and eye health business Bausch + Lomb, a revolutionary treatment called XIPERE (triamcinolone acetonide injectable suspension) was recently…
According to an article recently published in WhoTrades, Dupixent has recently been assessed in a clinical trial for the treatment of uncontrolled prurigo nodularis in adult patients. The data were…
In the past, psoriatic arthritis (PsA) has been relatively difficult to manage or control with current therapies such as biologic disease-modifying anti-rheumatic drugs (bDMARDs). While some patients respond well to…
According to a recent news release, the first cohort - consisting of healthy volunteers - was dosed in a Phase 1 clinical trial evaluating an intranasal formulation of 5-MeO-DMT. Ultimately,…
Previously, clinical-stage genetic medicines company Homology Medicines, Inc. ("Homology") submitted an Investigational New Drug (IND) application to the FDA for HMI-103, an investigational gene editing therapy designed to treat phenylketonuria…
The 37th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) was held last week from the 13th to the 15th. This event is the largest…
In June 2021, Redx Pharma dosed its first healthy volunteer in a Phase 1 clinical trial evaluating the safety and phamacological profile of RXC007. Altogether, this investigational therapy is designed…
Fast Track designation is requested by the drug company; this process and designation are designed to facilitate the development and review of therapies which can treat serious or underserved conditions,…
In the past, imatinib has shown promise for treating patients with pulmonary arterial hypertension (PAH). But specialty pharmaceutical company Tenax Therapeutics, Inc. ("Tenax") believed that it could create a specialty,…
Clinical trials can be helpful tools in determining the safety, efficacy, tolerability, recommended dose, and pharmacokinetics of specific drugs (among other things). In doing so, these trials help tell researchers…
Written by Lori Lawter, MPH We’ve heard a lot about bringing drugs to market lately, especially in light of the COVID-19 vaccine. You might wonder, “Is there a ‘secret sauce’…
Recently, biopharmaceutical company PTC Therapeutics, Inc. performed a five-year data analysis to evaluate the continued safety, efficacy, and tolerability of PTC-AADC. The company developed this novel gene therapy to improve…
The current standard-of-care for children with cerebral palsy often consists of a blend of occupational and physical therapy. But what if another therapy could be more beneficial? Medical XPress describes…
In the past, there have not been any available treatments to tackle cholestatic pruritus, or a severe and unrelenting itch, for patients with liver diseases such as Alagille syndrome. However,…
Clinical trials are important to understanding both more about certain conditions and more about the safety, efficacy, and tolerability of drugs designed to treat these conditions. Currently, biopharmaceutical company AMO…
Clinical trials have many endpoints - or milestones they are looking to achieve. These endpoints help signify the safety, efficacy, and tolerability of the treatments. In the Phase 3…
Unfortunately, clinical trials do not always proceed the way that drug developers - and patients - hope. For example, an ongoing Phase 1/2 clinical trial evaluating BMN 307, an investigational…
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