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…
The 59th Annual European Society for Paediatric Endocrinology (ESPE) Meeting is taking place virtually from September 22-26, 2021. During the Meeting, researchers are discussing best practices and treatments, as well…
According to an article submitted to StatNews by members of the Barth Syndrome Foundation, the FDA approved the drug Aduhelm in accordance with its accelerated approval program. Although the treatment…
Treatment for patients with GM2 Gangliosidosis - which consists of Tay-Sachs disease and Sandhoff disease - has often been symptomatic and supportive. However, a new therapy shows promise in benefiting…
In the past, Exelixis' drug CABOMETYX (cabozantinib) has been approved for use in a variety of patients. For example, the treatment is approved for patients with renal cell carcinoma (RCC)…
According to a recent article published in The Online Trading Mentor, Inventiva S.A. (Inventiva) has recently opened enrollment for its Phase 3 trial of Lanifibranor, an investigational treatment for nonalcoholic…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat patients with rare or life-threatening illnesses, defined as those affecting under 200,000 people or…
In the United States, Fast Track designation is granted to help expedite drug development and review for serious, rare, or life-threatening conditions. To receive Fast Track designation, the treatment must…
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