The safety, pharmacokinetics (absorption), and tolerability of the investigational eIF2B activator ABBV-CLS-7262 is being evaluated in a Phase 1b clinical study to treat patients with Vanishing White Matter (VWM) Disease.…
Omisirge (omidubicel) is the first allogeneic (from a donor) product for SCT that has been the recipient of FDA approval. The FDA's nod moves Omisirge into the realm of…
The National Organization for Rare Disorders (NORD), a Patient Worthy partner, formed in 1983 to provide support and education to people and families living with rare diseases, advocate for change…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
On April 25, 2023, myTomorrows, a platform designed to help patients obtain access to experimental medicines, recently launched a new tool called TrialSearch AI, an artificial intelligence-powered resource for physicians…
For many rare diseases, gene therapy, not traditional therapeutics, is perhaps the only means to significant relief and cure. However, the path to gene therapy has been strewn with obstacles…
Canada’s unemployment rate is at a record low. Industries have recovered 126% of the jobs lost by the pandemic. On the health front, scientific knowledge and the ability to…
Clinical trials are often mentioned as an afterthought when discussing the length of time it takes for drugs to be approved by the FDA. On the contrary, clinical trials are…
Prasanna Shirol, Executive Director of the Organization for Rare Diseases India (ORDI), expresses his opinion to the Times of India about the urgency to address the issue of rare…
There are still many unknowns when it comes to our genetics. How do gene variants of unknown significance (VUS) affect us? In what ways can we identify if variants…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
Collaboration has always made the world go round - and it's no different in rare disease research. Sometimes, the community affected by a certain rare disease is small, which…
On April 26th, I had the honor of speaking at the American Brain Foundation’s Commitment to Cures fundraising dinner during the American Academy of Neurology annual convention in Boston. I…
Altogether, there are an estimated 300,000 people in Ireland who are living with a rare disease. However, given the increasing amount of genetically-oriented rare diseases, it's possible that even…
Hello, my name is Megan. I have been diagnosed with postural orthostatic tachycardia syndrome (POTS), Ehlers-Danlos syndrome (EDS), mast cell activation syndrome (MCAS), small fiber neuropathy (SFN), eosinophilic esophagitis (EOE),…
On April 20, 2023, the Rare Disease Legislative Advocates (RDLA) hosted its monthly webinar. These webinars help provide updates to the rare disease community on legislation and other policy initiatives…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
STAT NEWS recently published an op-ed with a provocative question: How do we tell the difference between legitimate mental health tech and snake oil? Looking back to the early…
Over the years, there has been minimal improvement in the life expectancy differences between white and black people living in the United States. Yet, according to an article featured…
According to a recent study from McGill University in Canada, the new method for classification of brain illnesses has been identified. This study was led by Yashar Zeighami and compared…
The FDA has carried on a year-long disagreement with BrainStorm Cell Therapies, the developer of the experimental stem cell treatment called NurOwn. It is referred to as a personalized cell…
On February 1, 2023, FDA issued a Draft Guidance Document, called “Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products” (Docket ID: FDA-2022-D-2983). The Draft Guidance considers…
On April 12, 2023, Research!America, a Patient Worthy partner organization, hosted a webinar program titled Better Data for More Equitable Public Health Research. This talk featured Dr. Mary Pittman, President…
In 2003 Ricky Safer was diagnosed with primary sclerosing cholangitis (PSC). Her doctors were not able to tell her much about the origin of her disease. Nor were they able…
When Alexander Barron (9) was first born, doctors told his parents that he would likely never walk. In fact, said doctors, Alexander would probably not be very independent at all…
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