Normally, Fast Track is an FDA process designed to facilitate and expedite the development and review of drugs or biologics intended to treat rare or serious conditions. Recently, Healio…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
On August 17, 2021, Amnesty International released an urgent call to action to provide protection in Malawi for those with albinism. This call for change came following the death…
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…
Taylor Wilson, a woman from Buffalo, is raising money for hemophagocytic lymphohistiocytosis (HLH) in honor of her late friend Paige Casillo. The two have known each other since they were…
Unfortunately, while a variety of treatment options exist for different conditions, many come with unwanted adverse reactions or side effects. However, this might soon change for patients with HER2 positive…
Developing therapies for rare diseases can be difficult, and it often requires effort from many different players. Recently, AllStripes has made large steps forward in rare disease research by securing…
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…
On August 9, 2021, biopharmaceutical and RNAi therapeutics company Alnylam Pharmaceuticals, Inc. (“Alnylam”) shared that enrollment is now complete for the Phase 3 HELIOS-B clinical trial. During the trial, researchers…
In early August, via a press release, biotechnology company Athersys, Inc. shared that its Japanese partner, biotechnology company HEALIOS K.K. (“Healios”), recently released positive topline data from a medical study…
During clinical trials, the FDA may sometimes order a clinical hold. This order is often given to halt or suspend trial operations if the FDA believes that participants would…
In mid-August 2021, Parkinson's News Today reported that dosing was complete in a Phase 2a clinical trial. During the trial, researchers were evaluated ANVS401, an investigational compound for patients…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Science Magazine recently joined other news sources in reporting that Bluebird Bio’s Phase 3 clinical trial investigating a treatment for a neurological disease was put on hold by…
In early August 2021, the Icahn School of Medicine at Mount Sinai announced that it had received over $4M in grant funding from the National Cancer Institute (NCI). Mount…
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.…
Earlier this year, in June 2021, Guinea declared the end to the Ebola virus outbreak. However, just in case of a resurgence, some community health workers remained in Guinea…
In a news release from August 10, 2021, biotechnology company CohBar, Inc. shared that positive topline results were available from a Phase 1a/1b clinical trial evaluating CB4211 for patients…
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…
As recently presented in Rocketfacts. The thoracic outlet is a ring formed by ribs located immediately below the collarbone. Thoracic outlet syndrome (TOS) occurs when nerves or blood vessels…
The FDA has recently accepted Genentech's Biologics License Application (BLA) under priority review. This application was for faricimab, a bispecific antibody meant to treat ocular disorders, such as wet age-related…
Holden and Elliot have felt a surge of support from their community recently, as their preschool came together to celebrate them and show their love. Both boys were born with…
We're still learning about COVID-19 and its lasting effects, and we'll continue to do so for a long time. Researchers at Mount Sinai have been making progress, specifically with the…
It is important that we understand how medical conditions impact everyone, as knowledge helps with future research, drug development, and more. Unfortunately, medical research is not always inclusive. Because of…
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