A recent study has pointed to corneal collagen crosslinking (CXL) as a viable treatment option for children with keratoconus, a rare ocular disorder that impacts the cornea. As pediatric patients…
Poly ADP ribose polymerase (PARP) inhibitors are targeted cancer therapies which block PARP, an enzyme. Normally, PARP plays a role in DNA repair. Thus, inhibiting PARP prevents cancer cells from…
The month of May is recognized as Guillain-Barré syndrome (GBS) and chronic inflammatory demyelinating polyneuropathy (CIDP) awareness month, a time set aside to spread awareness about these rare conditions among…
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…
The month of May is recognized as Tuberous Sclerosis Complex Awareness Month, a time for helping to spread awareness about tuberous sclerosis complex (TSC) among the general public and in…
Anti-glomerular basement membrane disease (anti-GBM disease), formerly known as Goodpasture's syndrome, is a rare, autoimmune condition that impacts the kidneys and lungs. A recent study published in the Journal of…
Over the last 15~ months, the world has grappled with the impact of COVID-19, a novel coronavirus. Altogether, COVID-19 has led to over 156 million million cases with an associated…
The month of May is recognized as International Behçet's Awareness Month and May 20th is Behçet's Awareness Day. This is a special time for spreading awareness about Behçet's disease among…
There are two types of osteosarcoma, a rare bone cancer: localized or metastasized. If the cancer is localized, or in one area, the 5-year survival rate sits around 70-75%. However,…
In 2006, a new, rare disease affecting monochorionic twins was identified and named. Twin Anemia Polycythemia Sequence (TAPS) changed the perception of the complications of monochorionic twins. It started redefining…
19 seasons. 1,109 regular-season games. 35 playoff games. Multiple All-Star Games. This is the impressive resume of Tony Brown, a veteran referee for the National Basketball Association (NBA). Unfortunately, Brown…
Veterinary ophthalmologists at Pennsylvania’s Veterinarian School of Medicine found that LCA in humans mimics the same type of disease in dogs. According to a recent article in Science Daily, vision…
In a recent news release, gene therapy company Taysha Gene Therapies ("Taysha") shared the publication of preclinical data on the company's drug candidate, TSHA-102. Currently, Taysha is exploring TSHA-102 as…
In China, the Conditional Approval process allows for new drugs to be marketed and developed based on an urgent and unmet need. For example, drugs which treat patients with rare…
A recent study, titled "Patient and caregiver experiences of living with acute hepatic porphyria in the UK: a mixed method study," investigated the disease burden faced by AHP patients and…
Clinical trial data offers immense insight into a drug's safety, efficacy, and tolerability. But it can also share how combining two treatments can impact patient outcomes or whether a…
According to MedPage Today, data from a preliminary study evaluating SHR0302, an investigational Janus kinase 1 (JAK1) inhibitor, showed promise for treating patients with moderate-to-severe atopic dermatitis (AD). Following treatment,…
Patient-reported and patient-driven registries can be crucial in understanding rare diseases or cancers, driving research, and forming communities. Now, according to a recent press release, a new registry is forming,…
In a study published on Hindawi, a group of patients with severe cystic fibrosis that were awaiting a lung transplant underwent a telerehabilitation program to maintain their lung function. The…
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…
May 16 is recognized each year as HAE Day, a day dedicated to spreading awareness among the general public and the medical community about hereditary angioedema (HAE), a rare disease.…
There are many rare diseases that are not yet fully understood, with Parkinson's disease being one of them. Medical professionals have discovered a lot about the underlying causes of the…
As reported by CNN; Tabitha Haly still manages to make time for her life-long craft as a singer-songwriter and live performer while acting as a vice president at JP Morgan…
Professional musicians generally practice several hours every day, but that does not hold true for Reece Jacob, age 29, from Manchester. UK’s Daily Mail recently carried an article about Reece…
CureGRIN is investigating a new symptom of GRIN disorders, a group of rare genetic conditions that cause a wide variety of physical and intellectual symptoms. Referred to as neurostorms, this…
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