rare disease
2018 International Waldenstrom’s Macroglobulinemia Foundation Events You Don’t Want to Miss
After the 10th International Workshop on Waldenstrom’s Macroglobulinemia for researchers and clinicians, the IWWM10 International Doctor- Patient Forum will be held in the same...
International Symposium on Usher Syndrome and 10th Annual Usher Connections Conference
Put Europe on your calendar for 2018! Usher patients, researchers and family members welcome to the two-day Symposium in July. For more information, contact ush2018@usher-syndrome.org
International Alliance Of Patients’ Organizations – 8th Global Patients Congress –...
International Alliance Of Patients' Organizations hosts the 8th Global Patients Congress. 200 reps from different healthcare facets will provide expertise, connection, and knowledge related...
PBCers Organization – 2018 PBC Conference – Houston, Texas
PBCers Organization hosts the 2018 PBC Conference to connect, learn and empower. The event is catered to patients, and their family and friends to...
EURORDIS-Rare Diseases Europe’s European Conference on Rare Diseases and Orphan Products
“Rare Diseases 360° – Collaborative Strategies to Leave No-One Behind”
This event will be the largest rare disease themed conference in Europe. The conference will touch...
AKU Society – 2nd International Patient Workshop – Liverpool
AKU Society is hosting it's 2nd International Patient Workshop in Liverpool's Bluecoat. The event is intended to educate about new scientific research, clinical trials,...
Editor’s Choice: Enjoying Beauty Pageants, Family, and the Single Life with...
Happy Friday Everybody!
This week we're spotlighting three patient stories: an ALD patient full of optimism and love, a beauty pageant contestant with a noble...
The Value of Repurposing Old Drugs to Treat Rare Disease
According to a story from biotechconnectionbay.org, medical researchers are beginning to more closely investigate that possibilities that drug repurposing, or using old drugs for...
A Groundbreaking Marijuana-Derived Treatment is Getting Closer to FDA Approval for...
According to a story from statnews.com, a drug derived from substances originating in the cannabis plant may be nearing approval by the U.S. Food...
This New Cell and Gene Therapy Manufacturing Plant is the Largest...
The pharmaceutical company Lonza has just opened a new state-of-the-art manufacturing facility to produce cell and gene therapies. You can find the full press...
Survivors of Testicular Cancer May be at Greater Risk for Heart...
According to a story from Summit Medical Group, a recent study indicates that survivors of testicular cancer may have a greater risk for heart...
Direct to Customer Genetic Tests Could Make it Easier to Detect...
According to a story from wired.com, a new genomics marketplace called Helix will soon start selling genetic tests directly to consumers. These tests are...
After Life Saving Surgery, This Amyloidosis Patient is Spreading Awareness
According to a story from news.vanderbilt.edu, Charlotte Haffner has been working hard to advocating on behalf of amyloidosis patients. She recently underwent a heart...
Have You Heard of this Government Office Coordinating Rare Disease Research?
According to a story from Charcot Marie Tooth News, a small subdivision of the National Institutes of Health (NIH) is leaning into its role...
Experimental Tourette Syndrome Treatment Displays Potential in Trials So Far
According to a story from PR Newswire, the pharmaceutical company Therapix Biosciences recently announced the results of a Phase IIa study for its drug...
Spondyloarthritis Presents Differently in Kids
According to a story from medpagetoday.com, researchers are beginning to realize that symptoms of pediatric spondyloarthritis present themselves in a distinct way when compared...
Editor’s Choice: Get By with a Little Help from your Support...
Hope everybody is enjoying the warm weather!
This week we have patient stories about a woman with MS who started a support group and a...
Advocates for Rare Disease Patients Are Calling for a Patient Registry...
According to a story from QQ Malaysia Online, patient advocates are looking for ways to raise awareness of rare diseases in Malaysia and get...
Common Pain Killers May Not Worsen Ulcerative Colitis as Previously Thought
According to a story from scimex.org, a recent data review is calling into question the previously held consensus that common pain medicines such as...
8 Ehlers-Danlos Symptoms That Are Secretly Kind Of Fun
No matter which way you spin it, Ehlers-Danlos syndrome (EDS) is generally not a great time. EDS is a rare genetic condition that affects...
Patients with Ankylosing Spondylitis Don’t Fully Trust Biosimilars
According to a story from ankylosingspondylitisnews.com, a significant proportion of patients stopped taking a biosimilar for infliximab within six months of transitioning to the...
New Campaign Encourages Idiopathic Pulmonary Fibrosis Patients to Keep Exercising
According to a story from The Irish Times, a new campaign is encouraging people with the rare lung condition idiopathic pulmonary fibrosis to keep...
Patients with Mitochondrial Disease Are Having a Hard Time Getting Diagnosed
A recent story from Science Daily is highlighting just how difficult it can be for patients with rare diseases to get an accurate diagnosis....
A Physician Sees the Patient Perspective When Getting Treated For Primary...
According to a story from Neurology Advisor, a retired physician experienced the challenges of being a rare disease patient first hand after being diagnosed...
Treatment for Hereditary Angiodema Gets Marketing Approval From the EMA
According to a recent story from pm360online.com, the pharmaceutical company Shire announced that the marketing application for its drug product lanadelumab has gained approval...
