news Archives - Page 72 of 183

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The Department of Education’s 2020 Budget is Requesting Cuts to the Special Olympics and Special Education. What Does This Mean for Rare Patients?

Post author:James Moore
Post published:March 29, 2019
Post category:Rare Disease

If you are an avid follower of the latest news, then you have probably heard about one the latest sensational headlines to come out of DC lately: the US Department…

Development of New Imaging Technology Could Accelerate Parkinson’s Disease Research

Post author:Trudy Horsting
Post published:March 29, 2019
Post category:Parkinson's Disease Rare Disease

A recent generous donation from the Michael J. Fox Foundation for Parkinson's Research (MJFF) will help develop a novel piece of technology which could accelerate the research of new treatment…

Birth of Healthy Baby Monkey Indicates Promise of New Technique To Preserve Fertility of Childhood Cancer Patients

Post author:Trudy Horsting
Post published:March 29, 2019
Post category:Brain Cancer Soft Tissue Sarcoma Testicular Cancer

As the field of medicine has continually advanced, more and more children (now approximately 80%) are surviving cancer diagnoses. It's amazing. It also means that researchers are able to focus…

Updates in Experimental Therapies for Liver Disease

Post author:James Moore
Post published:March 29, 2019
Post category:Autoimmune Hepatitis Biliary tract cancer hepatic porphyria Rare Disease

According to a story from Healio, there are a number of recent reports of progress in the development of treatments for rare forms of liver disease. Many rare liver diseases…

Teen with Neuroblastoma has Made 90 Trips to the United States for Treatment

Post author:Trudy Horsting
Post published:March 28, 2019
Post category:Neuroblastoma

Robyn Smyth is a 14-year-old girl from Ireland who was diagnosed with neuroblastoma when she was three. Since then she's undergone countless rounds of chemotherapy, investigational treatments, a treasured period…

The Argument for a Second Independent Organization Dedicated Solely to Investigating Drug Safety

Post author:Trudy Horsting
Post published:March 28, 2019
Post category:Rare Disease

A recent article by Joe Graedon compares the FDA and the way the US investigates drug safety to the Federal Aviation Administration (FAA) and the National Transportation Safety Board (NTSB).…

New Potential Combination Therapy for Acute Myelogenous Leukemia has Received Breakthrough Therapy Designation

Post author:Trudy Horsting
Post published:March 28, 2019
Post category:Acute Myeloid Leukemia Rare Disease

Acute Myelogenous Leukemia Acute myelogenous leukemia (AML) is a rare blood cancer whose prevalence increases with age. There are still extremely limited treatment options for AML and the five-year survival…

Yet Another Study Shows the Dire Need for More Diversity in Medical Research

Post author:Trudy Horsting
Post published:March 28, 2019
Post category:Chronic Kidney Disease Huntington's disease Muscular Dystrophy

A recent study published in Cell has exhibited the scientific community's negligence for conducting research that includes an adequate representation of non-Europeans. Specifically, it showed that 78% of patients in genomic…

Formal Appeal to the CDC to Revise Incorrect Data Published on Lyme Disease

Post author:Trudy Horsting
Post published:March 28, 2019
Post category:Lyme Disease

Bruce Alan Fries, President of the Patient Centered Care Advocacy Group, along with Holly Ahern and Chris Fisk from the Lyme Action Network, have issued an official "Information Quality Request…

Study Gives Researchers Greater Understanding of Primary Progressive Aphasia

Post author:Trudy Horsting
Post published:March 28, 2019
Post category:Primary progressive aphasia Rare Disease

A preliminary study has found that dementia may only affect select areas of the brain in the early stages. Through this research, they've also uncovered what they believe is a…

Editor’s Choice: Approvals and Disappointments in Rare Research News

Post author:Patient Worthy Contributor
Post published:March 28, 2019
Post category:Rare Disease

Happy Thursday Everyone! We hope everyone's spring is off to a good start! This week, we're highlighting four articles about research updates. We have news pertaining to Methylmalonic Acidemia patients…

The Controversy of Noncompete Clauses and Their Effect on Rare Disease Patients

Post author:Trudy Horsting
Post published:March 27, 2019
Post category:Kidney Stones

The longer a patient sees a doctor, the more their initial trust, confidence, and comfort with them grows. This is amplified for patients living with rare conditions as doctors visits…

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New Combination Therapy for Multiple Myeloma Approved in England

Post author:James Moore
Post published:March 27, 2019
Post category:Multiple Myeloma

According to a story from Myeloma Research News, the UK National Institute of Health and Care Excellence (NICE) has recently decided to approve a three part combination treatment for coverage…

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Phase 3 Myelodysplastic Syndromes Trial Reaches Enrollment Milestone

Post author:James Moore
Post published:March 27, 2019
Post category:Myelodysplastic syndromes Rare Disease

According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Onconova Therapeutics, Inc. recently announced that they have successfully surpassed the 75 percent milestone for enrollment in the company's Phase 3…

Gaucher Disease Discovery Could Benefit Parkinson’s Disease Patients

Post author:Trudy Horsting
Post published:March 26, 2019
Post category:Gaucher Disease Parkinson's Disease

When one rare disease is found to be in some way similar to another rare disease, research can often be accelerated for both. For instance, recent discovery by a professor…

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Results from Three Clinical Trials for Urea Cycle Disorders and Cirrhosis Coming by the End of the Year

Post author:Trudy Horsting
Post published:March 26, 2019
Post category:Urea cycle disorders

Three non-investigational New Drug clinical studies are underway for Urea Cycle Disorders (UCD) and various forms of cirrhosis, initiated by Kaleido Biosciences. Dosing has been initiated in each of these…

Extremely Positive Results from Systemic Juvenile Idiopathic Arthritis 5-year Follow-Up Study in the EU

Post author:Trudy Horsting
Post published:March 26, 2019
Post category:Cryopyrin-Associated Periodic Syndromes Juvenile idiopathic arthritis Rheumatoid Arthritis

Extremely positive results have just been announced from a 5-year follow-up study of a systemic juvenile idiopathic arthritis (sJIA) treatment called Kineret. This treatment has been approved for use in…

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New Data From Phase 3 Cushing’s Syndrome Trial Presented

Post author:James Moore
Post published:March 26, 2019
Post category:Cushing Syndrome

According to a story from globenewswire.com, the biopharmaceutical company Strongbridge Biopharma plc, recently presnted a new analysis of data from the company's Phase 3 trial. This trial was testing the…

Investigational Farber Disease Therapy Now has Three FDA Designations

Post author:Trudy Horsting
Post published:March 26, 2019
Post category:Acid Ceramidase Deficiency Farber's disease Juvenile idiopathic arthritis

Farber Disease Farber Disease is a rare condition otherwise known as Acid Ceramidase Deficiency. It is a lysosomal storage disease caused by a missing ceramidase enzyme. This is due to…

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Two Companies Collaborate on Progressive Supranuclear Palsy Clinical Trial

Post author:James Moore
Post published:March 26, 2019
Post category:Progressive Supranuclear Palsy

According to a story from BioPortfolio, the biopharmaceutical company AlzProtect recently announced that PAREXEL Biotech, which is a subsidiary of PAREXEL International Corporation, will be expected to conduct a Phase…

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Phase III Ovarian Cancer Clinical Trial to be Discontinued

Post author:James Moore
Post published:March 26, 2019
Post category:Ovarian Cancer

According to a story from finanzen.ch, the drug companies Pfizer and Merck recently released a joint statement in which they announced that the companies' Phase III clinical trial would be…

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Study Indicates Safety of New Potential Treatment for Swallowing Dysfunction in Kennedy Disease

Post author:Trudy Horsting
Post published:March 22, 2019
Post category:Kennedy disease Spinal Muscular Atrophy

Kennedy disease or, Spinal and Bulbar Muscular Atrophy (SBMA) is a form of Spinal Muscular Atrophy (SMA). SBMA causes muscle weakness throughout the limbs which results in impaired mobility (typical…

Common Antifungal Drug Shows Promise in Treating Cystic Fibrosis

Post author:Trudy Horsting
Post published:March 22, 2019
Post category:Cystic Fibrosis

Cystic Fibrosis Cystic Fibrosis (CF) is a rare disease caused by a defected CFTR gene. It results in a buildup of mucus which impairs breathing and makes the lungs susceptible…

A Moebius Syndrome Story: An Unexpected Love that Crossed the Atlantic

Post author:Trudy Horsting
Post published:March 22, 2019
Post category:Moebius Syndrome

Alex Barker never planned on marrying someone with his same rare diagnosis, but he has to admit, the shared experience has made him and his wife closer. Alex has Moebius…

New Partnership will Improve and Accelerate Patient Involvement in Lung Cancer Research

Post author:Trudy Horsting
Post published:March 22, 2019
Post category:Adenocarcinoma of lung Anaplastic Lymphoma Kinase-Positive Non-Small Cell Lung Cancer

Pattern.Org Pattern.org was created by the Rare Cancer Research Foundation, a nonprofit dedicated to improving the lives of rare cancer patients by accelerating research. Pattern.org specifically, is a branch of…

The Department of Education’s 2020 Budget is Requesting Cuts to the Special Olympics and Special Education. What Does This Mean for Rare Patients?

Development of New Imaging Technology Could Accelerate Parkinson’s Disease Research

Birth of Healthy Baby Monkey Indicates Promise of New Technique To Preserve Fertility of Childhood Cancer Patients

Updates in Experimental Therapies for Liver Disease

Teen with Neuroblastoma has Made 90 Trips to the United States for Treatment

The Argument for a Second Independent Organization Dedicated Solely to Investigating Drug Safety

New Potential Combination Therapy for Acute Myelogenous Leukemia has Received Breakthrough Therapy Designation

Yet Another Study Shows the Dire Need for More Diversity in Medical Research

Formal Appeal to the CDC to Revise Incorrect Data Published on Lyme Disease

Study Gives Researchers Greater Understanding of Primary Progressive Aphasia

Editor’s Choice: Approvals and Disappointments in Rare Research News

The Controversy of Noncompete Clauses and Their Effect on Rare Disease Patients

New Combination Therapy for Multiple Myeloma Approved in England

Phase 3 Myelodysplastic Syndromes Trial Reaches Enrollment Milestone

Gaucher Disease Discovery Could Benefit Parkinson’s Disease Patients

Results from Three Clinical Trials for Urea Cycle Disorders and Cirrhosis Coming by the End of the Year

Extremely Positive Results from Systemic Juvenile Idiopathic Arthritis 5-year Follow-Up Study in the EU

New Data From Phase 3 Cushing’s Syndrome Trial Presented

Investigational Farber Disease Therapy Now has Three FDA Designations

Two Companies Collaborate on Progressive Supranuclear Palsy Clinical Trial

Phase III Ovarian Cancer Clinical Trial to be Discontinued

Study Indicates Safety of New Potential Treatment for Swallowing Dysfunction in Kennedy Disease

Common Antifungal Drug Shows Promise in Treating Cystic Fibrosis

A Moebius Syndrome Story: An Unexpected Love that Crossed the Atlantic

New Partnership will Improve and Accelerate Patient Involvement in Lung Cancer Research

The Let’s Chat CAR T One-on-One Mentor Program: Speaking with Someone Who Understands What You Are Going Through

“Shades of Psoriasis”: Plaque Psoriasis and Sharing Your Story

Understanding RSV: Why Combatting Ethnic and Age-Related Health Disparities Could Improve Outcomes

We Need Equity and Diversity Within Rare Disease Research

DNA in Color: How Ronya Nelson is Working to Close the Diversity Gap in Genetics - Part 1

Bridging the Diversity Gap in Healthcare

Congratulations to the 2023 Recipients of the Diversity in Lupus Research Awards!

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