The International Pain Foundation’s All in 1 iPain Summit
The All in 1 iPain Summit Los Angeles, CA #GetOnTrack This conference is dedicated to patients that are dealing with chronic, long term pain. This event will inform attendees about…
Kisaco Research’s Rare Disease Diagnostics Summit
Rare Disease Diagnostics Summit "Reducing the Diagnostic Odyssey to Improve Patient Outcomes" Boston, Massachusetts The diagnostic component of genetic rare diseases must be addressed. More than 350 million people are…
Cambridge Rare Disease Network’s RARESummit 2019
RARESummit 2019 Patients as Partners RARESummit 2019 is a rare disease conference unlike any other. The emphasis of this event is patient-centricity and partnership in the process of rare disease…
The Chinese Organization for Rare Disorders’ 8th China Rare Disease Summit
The Eighth China Rare Disease Summit The China Rare Disease Summit is the largest rare disease focused event held in China. A variety of rare disease stakeholders from across the…
Global Genes’ RARE Patient Advocacy Summit
The Global Genes RARE Patient Advocacy Summit This conference will be a convergence of a variety of rare disease stakeholders, such as rare disease patients, world renowned experts, and caregivers.…
.jpg)
Why Would Anyone Try to Compare Genetic Engineering to Weapons of Mass Destruction?
Why would someone compare genetic editing to weapons of mass destruction? Perhaps one answer can be found in the automobile. The family automobile was certainly not designed as a weapon…
<a href="https://pixabay.com/users/qimono/">qimono</a> / Pixabay
The Drug Firazyr is Safe for Older Hereditary Angioedema Patients, Study Finds
According to a story from Angioedema News, a recent study has found that the drug icatibant (marketed as Firazyr) can safely treat older patients with hereditary angioedema, a rare condition…
<a href="https://pixabay.com/users/geralt/">geralt</a> / Pixabay
This Mom Predicted Her Son’s Medulloblastoma Tumor Before The Doctors
According to a story from dailypost.co.uk, it didn't take long for Catherine Kelly to determine that her three year old son had a brain tumor; in fact, she made the…
<a href="https://pixabay.com/users/mwooten/">mwooten</a> / Pixabay
Grant From The American Neuromuscular Foundation Will Focus on Chronic Inflammatory Demyelinating Polyneuropathy
According to a story from newswise.com, the American Neuromuscular Foundation has recently awarded a grant to Dr. Karissa Gable. This grant will go towards a research project headed by Dr.…
<a href="https://pixabay.com/users/TheDigitalArtist/">TheDigitalArtist</a> / Pixabay
This Company Just Announced Plans to Develop a Gene Therapy for Type 2A Charcot-Marie-Tooth Disease
According to a story from BioSpace, the genetic medicines company Passage Bio recently announced plans for its sixth program for gene therapy development. The company plans to work with the…
Editor’s Choice: Studies on Studies on Studies
We're almost halfway through September believe it or not! As autumn approaches, we're continuing the aggregate rare disease news. This week, we're highlighting three studies, and one article on a…
<a href="https://pixabay.com/users/truthseeker08/">truthseeker08</a> / Pixabay
New Study Will Investigate Methods to Improve Cancer Trial Participation for Historically Under-Represented Groups
According to a story from journals.lww.com, The Lazarex Cancer Foundation and the University of California San Francisco have announced plans to work together on a three year study that will…
<a href="https://pixabay.com/users/blueberrykings111/">blueberrykings111</a> / Pixabay
An Experimental Drug Could be Effective for Neuromyelitis Optica, Study Results Show
According to a story from Biotech 365, the biotechnology company Viela Bio announced the publication of critical results from a recent study that tested the company's investigational drug inebilizumab as…
<a href="https://pixabay.com/users/Movidagrafica/">Movidagrafica</a> / Pixabay
Combined Clinics Improves Treatment for Autoimmune Diseases
According to a story from The Rheumatologist, evidence suggests that patients with rare diseases, such as autoimmune diseases, can benefit from visiting combined clinics, which are special medical centers in…
<a href="https://pixabay.com/users/Free-Photos/">Free-Photos</a> / Pixabay
In Canada, Medical Care is Covered. Now How Do You Afford Travel and Accommodations?
In the US, medical costs come in the form of drug prescriptions, doctors visits, expensive surgeries, and visits to specialists. As originally reported in The Tyee, in Canada, patients are…
<a href="https://pixabay.com/users/jarmoluk/">jarmoluk</a> / Pixabay
This Drug Could be a Breakthrough for Severe Familial Hypercholesterolemia, But How Much Will it Cost?
According to a story from Forbes, a new drug developed by the company Regeneron has the potential to make a real difference for people with the homozygous form of familial…
<a href="https://pixabay.com/users/katicaj/">katicaj</a> / Pixabay
A “Real World” Study Suggests That The Drug Apremilast Could be Useful for Behçet’s Disease
According to a story from rheumatology.medicinematters.com, a recent Italian real-world study has provided some evidence that the drug apremilast, which is considered a phosphodiesterase-4 inhibitor, could be a useful treatment…
<a href="https://pixabay.com/users/kmicican/">kmicican</a> / Pixabay
The Number One Health System in Virginia Sues Hundreds of Patients Per Week
According to a story from the Washington Post, the University of Virginia (UVA) Health System is often ranked as the best in the Old Dominion, but it also pursues debts…
<a href="https://pixabay.com/users/skeeze/">skeeze</a> / Pixabay
The All of Us Research Program is Recruiting Participants Steadily
According to a story from Columbia University, the country's All of Us Research Program is making consistent process in its ultimate goal to enroll a minimum of 1 million participants. These…
<a href="https://pixabay.com/users/PublicDomainPictures/">PublicDomainPictures</a> / Pixabay
This Single Genetic Alteration Could be Linked to Dozens of Medical Conditions
According to a story from Medical Xpress, a recent study has revealed that a certain genetic premutation affected the FMR1 gene could be a risk factor for far more health…
<a href="https://pixabay.com/users/Prawny/">Prawny</a> / Pixabay
Study Traces The Genetic Diversity of the Human Microbiome
According to a story from EurekAlert!, a team of scientists affiliated with the Harvard Medical School and the Joslin Diabetes Center recently published a study that is giving researchers the…
.png)
Patients in Mayo Clinic’s RIGHT Study Carried One or More Genetic Variants That May Affect Their Response to Medication
Pharmacogenomics plays an important role in predicting whether patients will benefit from certain medications or if the medication will cause serious side effects. The process involves the way in…
<a href="https://pixabay.com/users/geralt/">geralt</a> / Pixabay
For This Maroteaux-Lamy Syndrome Patient, Deportation Would Mean Certain Death
According to a story from Buzzfeed News, Maria Isabel Bueso was born in Guatemala with mucopolysaccharidosis VI, also known as Maroteaux-Lamy syndrome. At age seven she traveled to the US…
Attention Tyrosinemia Community Families: Come Together for Event in Late September
September is Tyrosinemia Awareness Month! The good people of the Network Of Tyrosinemia Advocates (N.O.T.A.) have organized a family weekend later this month aimed to bring the Tyrosinemia community together. Let's…
<a href="https://pixabay.com/users/PDPics/">PDPics</a> / Pixabay
2018 Saw Significant Improvements in Treating Rare Cancers
According to a story from curetoday.com, the American Society of Clinical Oncology (ASCO) recently announced its 2018 Advance of the Year. For 2018, the organization chose to highlight progress that…
