NLS Pharma has just announced new data which further supports the mechanism of action of Mazindol. Mazindol is the active ingredient in Quilience, a treatment used for narcolepsy. This therapy…
A recent study has found that Lambert-Eaton Myasthenic Syndrome (LEMS), a rare disease, could also be linked to another rare condition- Merkel cell carcinoma (MCC) which is a type of…
Connie Colbert, the Director of the Canyon Health and Wellness Clinic, describes what hyperhidrosis is, and what to do if you think you may have it. What is Hyperhidrosis? Hyperhidrosis…
Diabetes impacts many of the body's systems because it's an endocrine disorder. People with diabetes have trouble regulating their internal body temperature. As part of this, those with diabetes may…
Research has identified that individuals with sleep issues are at a higher risk for developing chronic diseases. Unfortunately, many with sleep disturbances are undiagnosed. It's estimated that between 40 and…
Michael E. Williams from the University of Virginia has just been awarded the Mantle Cell Lymphoma Leadership Award by the Lymphoma Research Foundation. This award represents not only his dedication…
New research has demonstrated the importance for understanding risks of bleeding, cardiovascular issues, nonadherence (due to the twice a day dosing), as well as gastrointestinal AE's when deciding to use…
Julie Anding can recall the exact moment of onset of her mal de debarquement (MdDS) symptoms. It was December 27th of 2017. She was in Utah on vacation for Christmas.…
The EMA has just recommended a combination therapy for patients with malignant pleural mesothelioma (MPM) that is considered unresectable. The treatment regimen includes nivolumab in combination with ipilimumab. This new…
A team of great researchers need your help! While the researchers who devised this survey (see below for the link) have had a great response from people with Mal de…
Quarantine during COVID-19 has been difficult for everyone. It's socially isolating, mentally draining, and physically limiting. We all find community different places. But no matter where we find it, it…
No one can truly prepare for life raising a child who has a rare disease. But as exhausting as it can be, Lori Dunham describes that raising her little girl…
Brickell Biotech has just announced the findings from their Phase 3 study investigating the long-term efficacy and safety of sofpironium bromide gel for the treatment of primary axillary hyperhidrosis. Hyperhidrosis…
The American Academy of Neurology recently held their annual meeting virtually to discuss the latest research in Dravet Syndrome (DS) and Lennox-Gastaut Syndrome (LGS). This meeting was held April 17th through 22nd,…
My name is Adam Nelson. I am 33 years old and I was born with a rare genetic connective tissue disorder known as epidermolysis bullosa simplex (EBS) of the Weber-Cockayne…
Growth hormone deficiency (GHD) influences patients both physically and mentally. The condition means patients' bodies don't produce enough growth hormone. As a result, they don't develop the same way as…
A study published in the Journal of Clinical Oncology has detailed the benefits of different forms of treatment for pediatric patients with adrenocortical carcinoma. These researchers particularly looked at what treatments…
Ryan Alam was diagnosed with mitochondrial-membrane protein-associated neurodegeneration (MPAN), a rare form of neurodegeneration with brain iron accumulation (NBIA), but he hasn't let these conditions slow him down. He has just…
Retinitis Pigmentosa Retinitis Pigmentosa is a rare condition which progressively causes vision loss. Genetic lesions which first affect the rod photoreceptors first cause vision loss at night. Next, cone photoreceptors are…
VISEN Pharmaceuticals has recently announced that enrollment has been completed for their Phase 3 clinical trial examining the safety and efficacy of TransCon hGH compared to daily rhGH in patients…
Nash Hensley is six years old, but in his short life he's already faced many challenges. He was diagnosed at birth with not one, but two rare diseases. First, Nash…
A recent study has found that thrombosis may be the first manifestation of granulomatosis with polyangiitis disease in children following the very first report of its presentation in a 13…
A recent study has found that aerobic and strength training exercise programs can have a long term effect on physical functioning for patients with pulmonary hypertension (PH). PH is a…
FibroGen has just announced that their investigative therapy pamrevlumab, for Duchenne muscular dystrophy (DMD), has received Rare Pediatric Disease designation from the FDA. The treatment already was given Fast Track…
C. difficile is a bacterial infection that impacts the gastrointestinal system. It can lead to weight loss, diarrhea, dehydration, and even the loss of blood platelets. Megan Newman has had…
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