Dana-Farber Cancer Institute has just announced research updates on multiple myeloma and waldenstrom macroglobulinemia which indicate significant progress in the search for treatments for these diseases. The Findings 1) There…
According to a story from pm360online.com, the biopharmaceutical company Ultragenyx Pharmaceutical Inc., in collaboration with Kyowa Hakko Kirin Co. Ltd, and Kyowa Kirin International PLC, recently announced that injectable burosumab…
According to a story from Medical Xpress, the US Food and Drug Administration has recently issued a new warning for the multiple sclerosis drug alemtuzumab (marketed as Lemtrada). This warning…
Most patients diagnosed with Homocystinuria (HCU), a rare metabolic disorder, don't receive their diagnosis until later in life. Scientists were at a loss for why patients with phenylketonuria (PKU), a similar…
This past Sunday, December 2nd, during the Patriot's game against the Minnesota Vikings, 20 players wore customized cleats to spread awareness for various philanthropic causes. This is the third year…
Phil Pavone owns a pawn shop in Norwich, Connecticut. It's called AZ Pawn. Now, in addition to being a businessman, he's practically created his own nonprofit. It all started one…
INSYS Therapeutics has just released data from their long-term safety study of CBD as a treatment for refractory pediatric epilepsy. The results were positive and we're excited about the future…
Chronic myelomonocytic leukemia, or CMML, is a rare condition. However, it has similarities to myelodysplastic syndromes (MDS) and myeloproliferative neoplasms (MPN). That being said, it still a unique diagnosis, and…
According to a story from BioSpace, the biotechnology company Tiziana Life Sciences Plc has recently announced that participant enrollment in its Phase 2a clinical trial has finished. This clinical trial…
Huntington's Disease is a neurodegenerative condition caused by a genetic mutation. It results in progressive decline of motor skills. It also affects cognitive function. Patients experience decline for 15-20 years…
Sickle cell disease (SCD) is a blood disorder. SCD patients have abnormal hemoglobin, or sickle hemoglobin, caused by a genetic mutation in the beta-chain. It results in deformed and inflexible…
Happy Wednesday! As the new month begins, we want to spotlight four articles. First, we have an incredible story about a Cushing's syndrome survivor and advocate and her talented service…
According to a story from einnews.com, the cell therapeutics company American Gene Technologies (AGT) has recently announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation…
In the UK, a think tank determines access to rare disease drugs based on their perception of an individual's quality of life. That idea has also been developed in the…
Amyotrophic lateral sclerosis (ALS) is a condition which affects the bodies nerve cells. It's neurodegenerative and progressive, causing the patient muscle weakness and muscle atrophy. It eventually results in paralysis.…
Mantle cell lymphoma is caused by a genetic mutation which produces cancerous white blood cells. Of course, these are the cells which fight infection in a normal body. Therefore in…
According to a story from BioPortfolio, the drug developer Novartis recently announced that the US Food and Drug Administration (FDA) has granted the company's investigational gene therapy AVXS-101, also known…
According to a story from BioPortfolio, the gene therapy company Rocket Pharmaceuticals has recently announced that the company's investigational gene therapy RP-L102 has earned both Fast Track and Regenerative Medicine…
According to a story from Reuters, the drug developer BioMarin is looking to make a name for itself in the hemophilia community in a very unique way. The company has…
According to a story from statnews.com, the recent FDA approval of a new treatment for Lambert-Eaton myasthenic syndrome could actually be bad news for patients. As it turns out, the…
Lambert-Eaton Myasthenic Syndrome (LEMS) is an autoimmune disorder. It's a rare condition characterized by muscle weakness in the limbs. When the muscle weakness includes the respiratory muscles, the disease can…
According to a story from Science Alert, an ugly and unpleasant tale involving the devastating rare genetic illness known as Huntington's disease will culminate in a lawsuit. The outcome of…
According to a story from Xconomy, the drug developer Vertex Pharmaceuticals recently announced that a three drug combination treatment for the rare lung disorder cystic fibrosis has performed well in…
On November 26, 2018, the FDA approved a new autoinjector for individuals with severe rheumatoid arthritis (RA), giant cell arteritis, and polyarticular or systemic juvenile idiopathic arthritis (aged 2 and older). It's…
On November 26, 2018, a new drug was given accelerated approval by the FDA. Not only that, but it was granted orphan drug designation and breakthrough therapy designation. The drug…
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