Welcome Back to Editor's Choice! The end of the week is finally here, and we have some reading material for you. First, we have an awesome story of a CF…
Comic Demetri Martin likes to point out that worms and bears don’t have much in common until you think about gummy. Then, the similarities become clear. What Martin is pointing…
These days there is an app for everything. There's an app for how to cook a nice meal. An app to remind you to meditate. Countless apps for finding your…
The United States' Orphan Drug Act (ODA) was enacted in 1983. Years later, it has failed to fuel the creation of orphan drugs to treat rare diseases, including spinal muscular…
When my son was born he didn't exhibit primary immunodeficiency (PI) or any of the other conditions on the newborn screening panel. Still, I fretted over just about everything. He…
Crinetics Pharmaceuticals, Inc. is a pharmaceutical/therapeutics company that focuses on rare endocrine-related disorders and cancers. This past month, excitingly, the pharmaceutical company announced that it had been awarded a Phase IIB…
July is the month of a lot of great things: The Fourth of July, long summer nights, fresh fruit, fireflies, and summer vacation. But there's something else great about this…
The pharmaceutical company, Kamada, has just submitted its proposed Phase III protocol for an inhaled alpha-1 antitrypsin therapy (inhaled AAT) to the U.S. Food and Drug Administration (FDA). This therapy…
It's getting hot out there... So you may as well read some news while staying cool inside if you're in the US! This week we have news on Senator John…
Rakesh Jain is a professor at the University of Texas Medical School, who recently spoke at a press conference for Neurocrine Biosciences. His talk was covered in an article published…
According to a recent study in Science Daily, scientists have found evidence that one treatment drug's effect on the cells for a specific condition could actually be applied to several…
Senator McCain has been a prisoner of war, has had a history of melanoma, and is 80 years old, so last Friday’s routine check-up didn’t end up being so routine.…
There’s no question that when life gets chaotic, your health is often impacted. It’s important to maintain a healthy balance for eating and living. Especially if you have hemochromatosis. Hemochromatosis…
We take most of our daily activities for granted. From working out at the gym to having our teeth cleaned, but for people living with hemophilia B what most people…
Happy Friday! How's the the sweltering heat treating you all (if you're on the east coast of the US, that is)? Hoping you are keeping cool! In the meantime, check out…
Great news for our friends in the the Gaucher community! The FDA and its counterpart across the pond, the European Medicines Agency (EMA), have outlined a new strategy to spur…
A recent Canadian study published in Seminars in Arthritis and Rheumatism announced findings that psoriatic arthritis disease activity showed trends of stabilizing and even improving during pregnancy. Psoriatic arthritis is…
A new study detailed in Nature, involving synthetically replicating certain cell activity has been hailed as a major breakthrough in developing therapies for many neurological disease – Parkinson’s chiefly among…
You have probably seen them, or at least heard of them. In fact, I’d go so far as to say you’ve seen them within the past week or two. The…
Patient Worthy attended the NIH‘s National Center for Advancing Translational Sciences NCATS meeting and in a word: WOW! Start with the patient- the end user, the source of the aberrant…
So much of it “comes down to morals.” A couple struggling with cystic fibrosis may have up to a 50% chance of passing the gene on to their child if…
Earlier last month, the second clinical trial for progeria was announced as completed. The trial was again funded by The Progeria Research Foundation, spearheaded by Drs. Scott Berns and Leslie…
Some good news for patients with Spinal Muscular Atrophy (SMA) and their families! There's a new booklet out with detailed information on how to navigate the increasingly complex health insurance…
As reported in Nature (an international medical publication), research scientists have uncovered the atomic makeup of an important protein found in those with Alzheimer's Disease. This is being touted as a…
Regular readers of Patient Worthy know the cost of drugs is a huge barrier to treatment for many patients and families. Additionally, the fewer people diagnosed with a condition, the…
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