Timber Pharmaceuticals focuses on rare diseases of dermatologic origin. These include scleroderma, congenital ichthyosis, tuberous sclerosis complex, and facial angiofibromas. They are currently working on two clinical trials. Trial 1…
CMT1A Charcot-Marie-Tooth disease (CMT) has a few different subtypes. 60% of all patients diagnosed with type 1 of the rare disease have CMT1A. This subtype means patients have an duplicate copy…
Recurrent Pericarditis Pericarditis is a rare autoinflammatory cardiovascular condition. It can lead to changes of the electrical conduction of the heart and a buildup of fluid surrounding the heart. Pericarditis…
Celltrion Healthcare has just announced positive progress for extending the authorization for the subcutaneous (SC) form of Remsima to five more autoimmune conditions. These are- Psoriatic arthritis Ankylosing spondylitis (AS)…
A new study published by ACS Nano and conducted by scientists at the University of Illinois at Urbana-Champaign uncovered a new way to modify stem cells. This study was conducted…
60% of all those diagnosed with multiple sclerosis (MS) also suffer from some form of sleep disorder. Fatigue is a more common symptom than even the well-known tingling and numbness…
Macular disease or stargardt disease, is a rare condition whose onset can occur at anytime. It results in damage to the retina, though most patients do not become completely blind.…
A recent study has demonstrated that through standardizing the whole genome sequencing process and making it routine for patients, we can improve rare disease diagnosis across the board. Earlier diagnosis…
Botox Botox was first approved as a treatment for strabismus and blepharospasm, rare eye conditions, in 1989. Now, it is approved for 11 different conditions including cervical dystonia, chronic migraines,…
By engineering exosomes, Sarepta Therapeutics believes they can utilize gene therapies, RNA therapies, and gene editing without triggering an immune response in the body. They have just announced a new…
A team of researchers recently evaluated a new biomarker in interstitial lung disease to see whether or not it could predict disease severity and ultimately mortality. The Biomarker Surfactant protein…
There is now a new version of the Neupro patch coming! This generic version was created by Vektor Pharma. Vektor concentrates on transdermal systems both through patches on the skin and…
International Nonalcoholic Steatohepatitis (NASH) Day takes place on June 12th every year. This year, a 4 part podcast series was released by NASHNET as part of the continued effort to spread…
Antigen-specific T-cell therapy is an innovative treatment option which uses immune cells to combat cancer cells. AdventHealth is the very first in the world to use this treatment for patients…
Leszek Ignatowicz, a researcher from Georgia State University, recently received a 5 year, almost 2 million dollar grant from the NIH for his investigation into multiple sclerosis (MS). This project…
Over 2018 and 2019, BioCryst Pharmaceuticals conducted two surveys to better understand the needs and expectations of hereditary angioedema (HAE) patients. Overall, they found that despite progress in HAE therapies,…
Myositis Myositis is an autoimmune disease that leads to muscle damage. Patients live with chronic disability due to the symptoms of this rare condition. These include skin rashes, pain, weakness, fatigue,…
A study conducted by researchers from Aix-Marseille University in France has revealed a new way to track disease progression of Charcot-Marie-Tooth disease type 1a (CMT1a)- changes to the nerves within the thigh.…
Celldex has just released positive results from a Phase 1 trial of an investigative therapy which could benefit patients living with chronic urticarias. This disease can result in decreased energy,…
International NASH Day, or IND, began in 2018 to raise awareness of nonalcoholic steatohepatitis (NASH). This is the most advanced form of fatty liver disease. It is a progressive condition…
Kimberly Underhay Kimberly Underhay is a Canadian who has survived cancer. She has also been diagnosed with osteoarthritis and has to use a walker. Her disabilities have left her unable…
Neurofibromatosis Neurofibromatosis is a rare condition that presents in different forms. Neurofibromatosis type one (NF1) causes tumors to grow on the plexiform neurofibromas, a type of nerve in the body. These tumors…
Lupus Nephritis Systemic lupus erythematosus (SLE) is a rare, chronic condition which affects a wide array of tissues in the body. A condition which signifies severe SLE progression is lupus…
IBDsmart Researchers at the University of Otago, led by Michael Shultz, have developed a new app called IBDsmart to help patients with ulcerative colitis or Crohn's disease receive care remotely.…
Jalee Pelissier lives with muscular dystrophy. When she was just 13 she became an advocate for others with disorders or disabilities like her own. Now 20, her advocacy has only…
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