On August 9, 2021, biopharmaceutical and RNAi therapeutics company Alnylam Pharmaceuticals, Inc. (“Alnylam”) shared that enrollment is now complete for the Phase 3 HELIOS-B clinical trial. During the trial, researchers…
Currently, there are no treatments available for patients with hereditary ATTR amyloidosis (hATTR); most available therapies are designed to aid with symptom management. However, biotechnology company and RNA-targeted therapeutics leader…
Amyloidosis is a rare disease which can cause organ failure. It is the result of an abnormal buildup of amyloid proteins in the blood. These proteins are then deposited within…
CRISPR is a gene-editing technology which holds the potential to treat a variety of genetic diseases and improve patient outcomes. Overall, CRISPR works by creating precise cuts and edits within…
Researchers have just announced that they have completed enrollment for a new Phase 3 trial for patients with transthyretin-mediated (ATTR) amyloidosis who have heart disease. This trial is examining a…
Currently, the European Hematology Association (EHA) Virtual Congress 2021 is going on from June 9 through 17, 2021. During the Congress, hematologists and other stakeholders will discuss clinical research and…
According to a story from globenewswire.com, the clinical company Prothena Corporation plc recently presented encouraging results from its phase 1 clinical trial. This trial was evaluating its investigational product candidate…
For more than 30 years, Ionis Pharmaceuticals, Inc. ("Ionis") has worked to create RNA-targeted therapies for patients in need. Now, according to a recent press release, the company initiated…
Throughout the COVID-19 pandemic, researchers have been dedicated to understanding which individuals are most at risk for severe disease. They've found that those who are immunocompromised and have certain cancers…
In early December 2020, many descended upon the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition to discuss insights in the field of hematology. During the Meeting, researchers…
According to a story from Newswise, the genome editing company Intellia Therapeutics has recently announced that the first patient has been dosed in its phase 1 trial, which will test…
In a recent press release, genome editing company Intellia Therapeutics, Inc. ("Intellia") announced its recent MHRA authorization to begin a Phase 1 clinical trial to evaluate its therapeutic candidate NTLA-2001. The…
According to a story from ajmc.com, a recent study found that a new combination treatment for systemic amyloid light-chain (AL) amyloidosis is more effective than the current standard treatment. The…
According to a story from PR Newswire, Akcea Therapeutics, Inc. recently announced that the Portuguese Medicine Regulatory Authority (INFARMED) has announced its approval of reimbursement for inotersen (marketed as TEGSEDI®)…
According to a story from Guru Focus, the biopharmaceutical company Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc. recently announced that the Ministry of Health of Spain has cleared…
According to a story from Biospace, the RNAi therapeutics company Alnylam Pharmaceuticals Inc. recently announced that its experimental product candidate vutrisiran has earned Fast Track designation from the US Food…
According to a press release from FirstWord Pharma, Gen and Alnylam Pharmaceuticals have signed an exclusive distribution deal in Turkey for RNAi therapeutic ONPATTRO. This therapy drug will treat…
According to a story from mdmag.com, a recent proof-of-concept style study suggests that a certain machine learning tool could be of benefit to patients living with the rare condition alkaptonuria.…
Christine Wright Christine Wright was an active, athletic, healthy mother of three. But in 2007 she started exhibiting strange symptoms that no one could give her an answer to. She…
As originally reported in Pfizer, the European Commission has just approved the first treatment option ever available to treat transthyretin amyloidosis cardiomyopathy (ATTR-CM). The medication, VYNDAQEL, taken orally once daily,…
Amyloidosis is a rare disease that has gone without awareness for a long time. This lack of awareness led to slow developments in treatment, issues with obtaining a diagnosis, and…
.jpg)
Pfizer issued a statement this week through its Media Relations that its drug VYNDAQEL® for treatment of Transthyretin Amyloid Cardiomyopathy received positive opinion by the European Medicines Agency’s (EMA) Committee for…
A big step forward for those with amyloidosis! The European Medicines Agency (EMA) granted Pfizer a positive recommendation for the approval of its treatment for amyloidosis, a rare disorder that…
Amyloidosis is characterized by stiffness and an inability to function in certain organs. Because of the danger this presents, it is important that doctors are able to monitor the…
.jpg)
A recent article in Bloomberg’s SFGate focused on the price of drugs. The average patient cannot afford the price of many drugs now on the market. Insurance companies have…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
