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An article published earlier this year by Fierce Biotech outlines an approach that was developed by researchers at the Fred Hutchinson Cancer Research Center. Using CRISPR-Cas9’s gene-editing method the researchers were…
The human spirit can be a remarkable force. Robb Freed cycled thousands of miles by himself to raise awareness for epidermolysis bullosa (EB) -- a rare illness that took the…
Treatment for ulcerative colitis differs based on the individual. Where the inflammation occurs, the severity, and the preference of the patient can all influence the treatment. For those with moderate…
TrialJectory is an organization working to match bladder, breast, colorectal, melanoma, myelodysplastic syndromes (MDS), and other cancer patients with the clinical trial that is right for them. They have successfully raised…
GlaxoSmithKline (GSK) has recently completed and released the results of Phase 3 of its study of benlysta. This drug is made for the treatment of lupus nephritis. Their BLISS-LN…
Savara Pharmaceuticals has recently interviewed Dr. Tisha Wang, who is a pulmonologist and critical care physician at UCLA. Dr. Wang is also an advocate for autoimmune pulmonary alveolar proteinosis…
Written by: Paul Pavao The most important thing I can tell you is that Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) can be survived. As of 2019 survival requires a bone…
Regeneron Pharmaceuticals has recently announced the results of Phase 2 of their study of pozelimab, which is an experimental treatment for paroxysmal nocturnal hemoglobinuria. Current therapies for this condition…
Genetic Counselors are an incredible resource – they can help assess your risk of developing a condition, the risk of your children inheriting a condition, and what potential treatment or…
No healthcare professional wants to hear that a drug being studied in a clinical trial did not meet the endpoints. It means that after countless hours of hard work,…
The FDA has just provided Orphan Drug Designation to Hizentra for the treatment of adults diagnosed with chronic inflammatory demyelinating polyneuropathy (CIPD). This is a maintenance therapy which works to…
Marinus Pharmaceuticals has recently announced updates on its ganaxolone programs for tuberous sclerosis complex (TSC), PCDH19-related epilepsy (PCDH19-RE), and CDKL5 deficiency disorder (CDD). Ganaxolone Ganaxolone is a positive allosteric modulator…
An open letter to The Editor from Gaetani et al was recently published in the Orphanet Journal of Rare Diseases. Dr. Gaetani and associates at the Fondazione Policlinico Universitario, Rome Italy,…
David Fajgenbaum was diagnosed with Castleman disease while he was still a medical student. He was perfectly healthy, but in a few days he had organs failing. He was treated…
A new study has been initiated by a digital health company in the United Kingdom called Congenica Ltd. This study focuses on re-annotating genes that may be associated with…
It is estimated that about 10% of people in the United States have rare diseases. But what makes a disease rare? How does this distinction affect diagnosis and treatment?…
Alpha-1 antitrypsin deficiency (A1AD) shares similarities with asthma, which leads to it often being misdiagnosed as asthma. This misdiagnosis leads to insufficient treatment. Toronto Western Hospital's Asthma and Airways…
According to a story from Targeted Oncology, the results of a recent clinical trial have left drug developers Kite and Gilead with the confidence to proceed with regulatory approval from…
Karyopharm, a pharmaceutical company based in Newton, Massachusetts, has submitted a New Drug Application to the FDA for their drug XPOVIO, which is meant for the treatment of diffuse large…
Prevail Therapeutics recently submitted an Investigational New Drug application for their gene therapy called PR001. The FDA has recently notified them that this application is active, meaning that Prevail Therapeutics…
Copyscape score: 4% Cancer is a disease that affects different people in different ways, and for some people, no existing treatments can effectively treat their cancer. GlaxoSmithKline has recently released…
Copyscape score: 2% Alnylam has recently completed the third phase of their clinical trial of lumasiran, which was created for the treatment of primary hyperoxaluria type 1 (PH1). While they…
In the world of rare diseases, progress can often feel like a slow and agonizing slog. In many cases, this is the unfortunate reality. However, a lot can change in…
If you are unable to work because of a medical condition, you may qualify for disability benefits from the Social Security Administration (SSA). The SSA oversees two different disability programs, Social Security Disability…
The EMA has just announced that Danicopan has been accepted into its PRIority MEdicines (PRIME) program. Danicopan is being developed for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood condition. PRIME…
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