Tafenoquine is an anti-malarial drug that interferes with parasitic growth in the body. But could it be repurposed for use in another parasitic illness? That's what pharmaceutical company 60 Degrees…
People with idiopathic pulmonary fibrosis (IPF) already have two FDA-approved treatment options: Ofev (nintedanib) and Esbriet (pirfenidone). But biotech company Agomab Therapeutics ("Agomab") is working to introduce another potential therapeutic…
In the United States, Orphan Drug designation is granted by the FDA to drugs or biologics designed to treat, diagnose, or prevent a rare condition affecting fewer than 200,000 people…
Historically, it has been difficult to incentivize companies to develop therapies for rare or “orphan” conditions. The National Conference of State Legislatures (NCSL) explains that “orphan” conditions are: neglected conditions…
Right now, no cure exists for people living with systemic sclerosis (also known as systemic scleroderma). There are treatment options available to manage systems. However, these also come with issues.…
Prader-Willi syndrome (PWS) is a rare genetic disorder with a number of physical, emotional, mental, and behavioral manifestations. Individuals with PWS may exhibit cognitive impairment, hypotonia (poor muscle tone), and…
Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…
Developing novel therapeutics for rare diseases is increasingly important. Understanding the unmet needs of rare disease communities and providing effective, accessible therapies can significantly improve the lives of those affected.…
The research landscape for Duchenne muscular dystrophy (DMD) has been steadily increasing. Researchers are exploring more therapies, including gene therapies, that could transform the lives of those with this rare…
In mid-January 2023, Kristi Rosa of OncLive reported that the European Medicines Agency (EMA) granted Orphan Drug designation to pimicotinib (ABSK021) for inoperable tenosynovial giant cell tumors (TGCT). Orphan Drug…
In the European Union, Orphan drug designation is granted by the European Commission (EC) to drugs or biologics that are intended to diagnose, prevent, or treat rare conditions. The definition…
An estimated 90% of the global population is infected with Epstein-Barr virus (EBV). In some cases, this infection is asymptomatic; in others, EBV can lead to numerous health impacts, including…
Healio Psoriatic Disease recently reported that Vyjuvek (beremagene geperpavec-svdt), a topical gene therapy designed for people with dystrophic epidermolysis bullosa (DEB), was granted Orphan Drug designation (ODD) by the Japanese…
Historically, drug development within the rare disease space has been stifled; companies tend to focus on developing therapies for bigger patient populations that will be more impactful and more…
In the United States, Orphan Drug designation is granted by the U.S. FDA to therapies or biologics intended to treat, diagnose, or prevent rare conditions affecting fewer than 200,000…
Right now, there is an urgent need to identify and develop treatments for people with malignant gliomas, including glioblastoma. Unfortunately, there are limited effective treatment options; these cancers are…
Currently, there are no FDA-approved treatments designed for cognitive impairment related to Huntington’s disease. However, to improve the lives of those affected, additional research and therapeutic development are urgently…
Historically, it has been challenging to mobilize drug development and research within the rare disease sphere. Given the smaller community sizes, as well as a general lack of profitability,…
In July 2023, the U.S. FDA granted Orphan Drug designation to ABM-1310, an investigational treatment option for people living with malignant gliomas such as glioblastoma. Now, reports clinical-stage biopharmaceutical…
Setanaxib is a NOX enzyme inhibitor initially developed by Calliditas Therapeutics AB (“Calliditas”) for the treatment of primary biliary cholangitis. NOX enzymes produce reactive oxygen species (ROS) in the…
As many people in the rare disease community know, it can be difficult at times to galvanize support for improved drug development. Many larger pharmaceutical companies may be hesitant to…
370 words (source - 3% match) vs. 496 words (mine - 2% match) GEENIE can be used to develop in vitro and in vivo delivery mechanisms for drugs, gene editing…
In the United States, the FDA grants Orphan Drug designation to drugs or biologics that are intended to treat, diagnose, or prevent rare conditions. A rare condition is one…
245 words (source - 6% match) vs. 386 words (mine - 4% match) In the European Union, Orphan designation is granted to therapies intended to treat or use for a…
Previously approved for the treatment of narcolepsy, pitolisant (marketed under the brand name Wakix) may soon be leveraged for another rare condition: idiopathic hypersomnia (IH). Pitolisant is a histamine 3…
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