In a recent press release, biotech company AltruBio Inc. shared that its therapy neihulizumab (AbGn-168H) received Fast Track designation for the treatment of patients with steroid refractory acute graft-versus-host disease…
Being diagnosed with a rare disease can bring on various emotions; everybody's experience is unique to them. Some feel relieved to finally put a name to their symptoms; others are…
Kimberly Warner Kimberly Warner was diagnosed with mal de débarquement syndrome (MdDS) after enduring a traumatic biking accident in May of 2014. The accident caused her to break her pelvis…
Biopharmaceutical company OncoSynergy recently shared that the first patient was dosed in a Phase 1 clinical trial. Within this trial, researchers are exploring OS2966 for patients with recurrent glioblastoma.…
According to a story from Valdosta Daily Times, a collaboration between the biotechnology company ReflectionBio® and the nonprofit organization Invincible Vision is taking aim at the rare disease Bietti's crystalline…
According to a story from bloomberg.com, the biopharmaceutical company Rallybio has announced two development programs for therapeutics intended to treat rare diseases. The two programs consist of RLBY212, which is…
In a recent press release, the Ivy Brain Tumor Center, a nonprofit translational science initiative, shared that the first patient was treated in a Phase 0 clinical trial. The clinical…
Aljazeera recently published an article that highlights the difficulty several families have had waiting for a rare disease diagnosis. In the majority of cases, when the families finally received the…
According to Healio, giant-cell arteritis (GCA) is seeing a worrying trend. Over a period of 18 years (2000-2018), mortality rates for GCA increased from 50 to 57.6 per 1,000 deaths.…
Suddenly Sleepy Saturday, a day dedicated to spreading awareness about narcolepsy, is being held on March 13, 2021. The Narcolepsy Network is looking for volunteers to send a proclamation request…
At the end of February 2021, BridgeBio Pharma Inc. ("BridgeBio") and its affiliate Origin Biosciences, Inc. ("Origin") shared that its treatment, NULIBRY (fosdenopterin) for Injection, received FDA approval for patients…
March is recognized as Myeloma Action Month, a time dedicated to spreading awareness among the general public and the medical community about multiple myeloma, a rare hematological cancer. This event…
Pharnext has released an update on the clinical development of PXT3003, a treatment for Charcot-Marie-Tooth disease type 1A (CMT1A). According to the biopharmaceutical company, they are on track to dose…
A recent article in the journal Nature describes the first draft of human genome analysis and sequencing published in 2001 by the Human Genome Project international consortium. The consortium included…
Parker Boyer and his family know many of the obstacles that rare disease patients face; they've encountered many of them since Parker's birth. In fact, they dealt with a number…
According to a recent article featured in News5Cleveland.com, there are over seven thousand rare diseases worldwide that affect over thirty million people in the United States. Several days ago, 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…
Evotec SE and Chinook Therapeutics have entered into a strategic partnership in an effort to find, create, and develop viable treatments for those living with chronic kidney diseases. Under the…
If you've ever looked into the field of medical research, you may have heard the term biomarker. In short, a biomarker is a type of measurable sign or indicator which suggests…
When a patient is told that they have a life-threatening rare disease and then told that there is no cure, every effort is made to attend to their physical needs.…
The Orange County Guide features doctors’ articles in each issue. Dr. Justin West’s search to cure his son’s rare disease will be familiar to families also in search of…
Vanderbilt University has just announced a new collaborative agreement with Soleno Therapeutics in aim to develop KATP channel activators as a therapeutic for rare diseases. This therapy has already been…
As reported in News Wise, these oncologists don’t like their options. They want to provide patients with the best possible options to tackle the lethal cancer mesothelioma, but their hands…
Together, Takeda Pharmaceuticals and the Children's National Hospital have created a program that aims to create and improve a standard of care for rare disease patients. As these patients often…
According to a story from Market Watch, the gene therapy company AVROBIO, Inc. has recently announced that its investigational gene therapy candidate AVR-RD-04 has earned Orphan Drug designation from the…
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