There is no cure for multiple sclerosis (MS). However, treatment advances have helped people to live longer, happier lives. Current therapeutic options include corticosteroids, plasmapheresis, and disease-modifying drugs. Recently,…
In 2020, the U.S. FDA granted Orphan Drug designation to KB407, an investigational gene therapy for patients with cystic fibrosis (CF). More recently, at the start of 2023, the…
According to a story from the Rancho Santa Fe Review, 26-year-old Tyler Schutz began taking the sport of golf by storm last year. The young man made the Double Eagle…
According to a story from the East Bay Times, it was 2003 when Isabel Bueso and her family traveled to the US from Guatemala. She was just eight years old,…
In the European Union (EU), Orphan designation, or Orphan Medicinal Product designation, is granted to drugs or biologics intending to treat, diagnose, or prevent a rare, chronically debilitating, or…
Singer Celine Dion recently acknowledged that she has an extremely rare neurological disorder called stiff person syndrome (SPS). According to a recent article in The Metro, the disease occurs…
According to a story from The Post and Courier, Chantel Bass and her family have been living in the shadow of a rare immune illness called chronic granulomatous disease (CGD).…
New Delhi: Recent headlines in the Hindustan Times highlighted a letter written by the leader of the Bharatiya Janata Party, Varnum Gandhi, to Health Minister Mansukh Mandaviya urging him…
On January 6, 2023, the FDA granted conditional approval to a new Alzheimer’s drug, lecanemab, that will be sold as Leqembi. Vox carried the original story and included an…
Rare disease stakeholders are called on to participate in the first Rare Disease Therapeutic Alliance of 2023 featuring an agenda presented by Rare Advocacy Movement experts. The agenda will…
423 words 10% matched vs 524 words 5% matched The Rare Diseases International Organization reports that the groundbreaking UN Resolution on Persons Living With a Rare Disease (PLWRD), effective…
One year ago, in December 2021, clinical studies of lovo-cel for patients who were eighteen or younger were put on partial hold by the FDA. At the time, bluebird…
Orphan Drug designation is granted by the FDA to drugs or biologics intended to treat, diagnose, or prevent a rare disease or condition. “Rare,” in the United States, is…
Throughout his life, Gavin Miller has overcome many struggles. As a young child, Gavin always loved hockey. But when he was in a hockey program at age four, he…
On December 23, 2022, President Joe Biden officially signed the Consolidated Appropriations Act, 2023. This bill sets in stone the federal government's budget for the year, so naturally it includes…
When the COVID-19 pandemic swept the world, nobody knew what the short- or long-term effects would be. Now, a few years into the pandemic, we’ve begun understanding some of…
According to a story from empr.com, the Oncologic Drugs Advisory Committee, part of the US Food and Drug Administration (FDA), recently voted 16 to 0 against the investigational drug omburtamab…
In May 2016, I was diagnosed with a rare genetic neurological disease called adrenoleukodystrophy (ALD). Sadly, it's incurable and there's no medication to ease my condition. Since then, my life…
According to a story from PR Newswire, the biopharmaceutical company AMO Pharma Limited has recently announced the completion of enrollment for its REACH-CDM clinical trial. This trial will be investigating…
For those of us who suffer from depression, that mostly invisible mental illness that, at its worst, can render us incapable of getting out of bed and, at its best,…
According to a story from PR Newswire, the cell therapy company Cellevolve Bio, Inc., recently announced that the US Food and Drug Administration (FDA) has advanced the development of its…
In 2021, Alyssa, who is now thirteen years old, and her family were told that she had acute lymphoblastic leukemia. Alyssa did not respond to either the standard leukemia…
Currently, the standards-of-care for advanced/metastatic (stage III or IV) melanoma include immunotherapy, targeted therapy, and radiation. Some patients are given anti-PD-1 inhibitors. However, only around 20-30% of those living…
According to a press release published on globalgenes.org, the US Food and Drug Administration (FDA) has expanded the label for Oxlumo, a drug developed by Alnylam Pharmaceuticals as a treatment…
At the moment, there are two FDA-approved treatment options for people with idiopathic pulmonary fibrosis (IPF). However, given the needs of patients, it is necessary to develop additional therapeutic…
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