by Lauren Taylor from In The Cloud Copy Spinal muscular atrophy or SMA is a genetic disease that affects the central and peripheral nervous systems, as well as voluntary muscle…
According to a story from GlobeNewswire, Exagen, Inc. recently launched a testing panel called AVISE® Vasculitis AAV, which is designed to help physicians get fast and accurate data for patients…
For many years, researchers linked obesity to a number of other health conditions. Now, Taiwanese researchers identify a new cause for concern: worsening health outcomes for patients with ankylosing…
In a recent press release, clinical-stage pharmaceutical company Crinetics Pharmaceuticals announced that their investigational drug candidate, CRN04777, received Rare Pediatric Disease designation from the FDA. The therapy is designed…
Disclaimer: This blog post has been republished with permission from the author. To visit the original blog site, click here. Written by Tamara Fowler Where do I begin? Thank you…
Happy Friday! September is a packed with rare disease awareness, as we've been featuring all month long. We wanted to make sure to draw attention to Dystonia Awareness Month, which…
In a recent press release, precision therapy company Blueprint Medicines Corporation announced positive results from two clinical trials: EXPLORER and PATHFINDER. The trials examined the safety, efficacy, and tolerability…
Knowing how to help treat your disease and being able to afford those needs are two different questions. As reported in Newswise, in a study from Harvey L. Neiman Health Policy Institute, researchers…
As sourced from BioSpace: After 44 years of the legendary Jerry Lewis hosting the Jerry Lewis MDA Telethon to bring awareness to this disorder, his tradition will live on in…
CRISPR Therapeutics, in combination with Vertex Pharmaceuticals, has recently announced that the European Medicines Agency (EMA) has granted the Priority Medicines (PRIME) designation for their sickle cell disease (SCD) treatment,…
by Lauren Taylor from In The Cloud Copy Rheumatoid arthritis (RA) is an auto-immune condition that occurs when the body’s immune system mistakenly targets the joint linings causing inflammation, pain,…
According to a story from Charcot-Marie-Tooth News, an experimental HDAC6 inhibitor called CKD-504 appeared to restore myelination in mouse and cell models of Charcot-Marie-Tooth disease type 1A in a recent…
Dr. Naveen Pemmaraju recently interviewed with Targeted Oncology and discussed the many advances in myeloproliferative neoplasms (MPNs) treatment as well as the role of social media during the pandemic.…
Chronic pain is a common problem in the United States; it is estimated that 25.3 million people in the US experience chronic, severe pain every day. Within these millions of…
Disclaimer: This blog post has been republished with permission from the author. To visit the original blog site, click here. Written by Tamara Fowler The weekend of June 11, 2011,…
Turning 18 means many things: the right to vote, buy a lottery ticket, join the military, and even go skydiving. That transition into independence gives freedom, but also newfound responsibility.…
According to a recent article in Business Wire, it is estimated that five million people in the United States over the age of sixty-five are coping with Alzheimer’s at…
According to a story from Medpage Today, the Society of Hematologic Oncology recently held its virtual meeting on September 9-12. During the event, Dr. Srdan Verstovsek of the MD Anderson…
Dr. Richard Furie at New York’s Feinstein Institutes recently interviewed with MedPage Today. Dr. Furie discussed the prognosis for lupus nephritis and belimumab (Benlysta), a recently-tested drug that appears…
Did you know that testing protein levels in our bodies can tell researchers about our health? In some cases, it can even give insight into disease progression and development.…
This year, thanks to COVID-19, many conferences have been held online. These play a crucial role in spreading data, facilitating conversations, and determining potential treatment options for patients with…
I'm sure you have heard about Zika virus before, but what about Chikungunya? Both are rare mosquito-borne diseases that can cause severe neurological problems. But now, researchers also believe…
According to a story from BioSpace, the biopharmaceutical company Bristol Myers Squibb Canada recently announced that its medication fedratinib (marketed as INREBIC®) has been approved by Health Canada as a…
A new treatment for acromegaly has recently become available for patients. Called MYCAPSSA, this delayed-release capsule is intended for long term maintenance of acromegaly. Considering that injections are the typical…
The care of a loved one with a debilitating condition often falls to their mother, but fathers, siblings or spouses also may take on that role. Even if there is…
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.
