Patient Worthy Contributor – Page 39

Amy’s Acromegaly Story: Part 1

Post author:Patient Worthy Contributor
Post published:February 12, 2018
Post category:Acromegaly Rare Disease

My name is Amy, and I’m a 37 year old single mom of two beautiful girls, ages 8 and 13. I work full time as a business analyst for a…

Editor’s Choice: When Sandwiches and Air Horns Change Lives

Post author:Patient Worthy Contributor
Post published:February 9, 2018
Post category:Rare Disease

Welcome back, Patient Worthians! This week, we have an opportunity for Parkinson's patients, and an update on an Amyloidosis app. We also have two stories of teenagers whose lives suddenly…

Cognitive Impairment, Testing, and Disease Burden in MPS II Discussed at Satellite Symposium

Post author:Patient Worthy Contributor
Post published:February 7, 2018
Post category:MPS II (Hunter Syndrome)Rare Disease

Today, Professor Elsa Shapiro and Dr. Christina Lampe presented "Cognitive Impairment in Patients with MPS II: From Disease Burden to Cognitive Testing" at Satellite Symposium, an event sponsored by Shire…

Source: Pixabay

New Study Shows Mice Can Successfully Mimic Human Cancers

Post author:Patient Worthy Contributor
Post published:February 7, 2018
Post category:BRCA-mutated Breast Cancer Rare Disease

A recent study from Michigan State University confirms that mice do indeed have significant similarities to humans which is beneficial for cancer research, reported News Wise. This has been in…

qimono / Pixabay

Satellite Symposium Discusses Long-Term Outcomes for MPS IVA and VI Patients

Post author:Patient Worthy Contributor
Post published:February 7, 2018
Post category:MPS IV (Morquio syndrome)MPS VI (Maroteaux-Lamy syndrome)Mucopolysaccharidosis

Yesterday, a group of experts presented "Changing lives: Long-term outcomes of MPS IVA & VI patients" at the Satellite Symposium sponsored by BioMarin. The program began with insights on the…

Editor’s Choice: Songs, Viruses, and Legal Battles

Post author:Patient Worthy Contributor
Post published:February 2, 2018
Post category:Rare Disease

Happy Groundhog Day, Patient Worthians! It might not feel like it, but spring is its way! This week, we have a story of a family who copes with the challenges…

Editor’s Choice: Just Keep Swimming

Post author:Patient Worthy Contributor
Post published:January 26, 2018
Post category:Rare Disease

Happy Friday Patient Worthians! Believe it or not, we've almost made it through the first month of 2018! This week, we have a story of a young girl with Sanfillipo…

Editor’s Choice: Treatment Wishes Really Do Come True

Post author:Patient Worthy Contributor
Post published:January 19, 2018
Post category:Rare Disease

TGIF, Patient Worthians! This week, kids went to bed wearing inside out pajamas and wishing for snow. Their wishes came true-- and so did the wishes of many rare disease…

Source: Pixabay

Braving the Storm: How Music and Family Helped Tara Get Through a Tough, Rare, Little-Known Disease

Post author:Patient Worthy Contributor
Post published:January 19, 2018
Post category:Mast Cell Activation Syndrome Rare Disease

I know you’re fighting hard//I see your battle scars It’s clear you’ve come so far//You’ll grow stronger Alone, these words may feel like just expressions of encouragement; a…

Editor’s Choice: When it Comes to Rare Diseases, Unity is Strength

Post author:Patient Worthy Contributor
Post published:January 12, 2018
Post category:CGD Rare Disease

Happy Friday, Patient Worthians! This week we have articles about people working together on different types of rare disease teams. We have stories from a family who supports two children…

How to Survive and Thrive: Tips From an LGS Mother

Post author:Patient Worthy Contributor
Post published:January 11, 2018
Post category:Lennox-Gastaut syndrome Rare Disease

When you are in the midst of difficult times in your life, what do you do to make it through? Do you try and find a way to hide out…

Source: Pixabay

Here’s How You Can Help the Charcot-Marie-Tooth Association!

Post author:Patient Worthy Contributor
Post published:January 11, 2018
Post category:Charcot-Marie-Tooth disease

Patient Worthy recieved a note from CMT Warrior and Patient Worthy Contributor Quentin Martin of a worthwhile cause, that we think all our readers should know about! Quentin writes: If…

Retrophin Gift to Establish Rare Disease Network at Children’s Hospital

Post author:Patient Worthy Contributor
Post published:January 9, 2018
Post category:Rare Disease

WASHINGTON, D.C. & SAN DIEGO — Children’s National Health System and Retrophin, Inc. (NASDAQ: RTRX) are pleased to announce the creation of the Retrophin Rare Disease Network at Children’s National.…

Editor’s Choice: Ringing in the 2018 with Awareness

Post author:Patient Worthy Contributor
Post published:January 5, 2018
Post category:CGD Rare Disease

Welcome back, Patient Worthians! We're starting out the new year with stories of strength and awareness. This week, we have an article about a man with CSF leak, whose life…

Source: Pixabay

Orphan Drug Designation Granted for New IPF Treatment

Post author:Patient Worthy Contributor
Post published:January 3, 2018
Post category:Idiopathic Pulmonary Fibrosis Rare Disease

Good news for Prometic Life Science new drug application! The pharmaceutical company has been searching for a cure for idiopathic pulmonary fibrosis (IPF), a rare lung disease which you can…

Editor’s Choice: New Year, New Treatments

Post author:Patient Worthy Contributor
Post published:December 29, 2017
Post category:CGD Rare Disease

Happy New Years, Patient Worthians! New year, new you... new treatment news? This week, we have an article about a molecule that could change the way we treat cystic fibrosis.…

pixabay.com

Disappointing News for Uveitis Treatment

Post author:Patient Worthy Contributor
Post published:December 29, 2017
Post category:Uveitis

Pharmaceutical company, Santen, has been developing a drug, intravitreal sirolimus, to treat noninfectious posterior segment uveitis. Uveitis is a type of eye inflammation, in which the uvea, which is the middle part…

Source: Pixabay

How Gut Bacteria Could Change Your Response to Renal Cell Carcinoma Treatment

Post author:Patient Worthy Contributor
Post published:December 29, 2017
Post category:Rare Disease Renal Cell Carcinoma

The more scientists learn about gut bacteria, the more fascinating it becomes. It can affect your mood, the foods you crave, and according two new studies, it can even change…

Source: Pixabay

New Trial for sGC Stimulator Treatment for Sickle Cell Disease

Post author:Patient Worthy Contributor
Post published:December 29, 2017
Post category:Sickle cell anemia

Sickle cell disease causes red blood cells to form abnormally, which disrupts oxygen function. Cancer Prevention Pharmaceuticals have been working with Vanderbilt Medical Center and NCI to start a new Phase…

AndCan / Pixabay

The Monoclonal Antibodies That Could Treat Hepatocellular Carcinoma

Post author:Patient Worthy Contributor
Post published:December 26, 2017
Post category:Hepatocellular Carcinoma Neuroblastoma Thyroid Cancer

Hepatocellular carcinoma is a form of cancer that starts out in the liver. As far as cancers that begin in the liver, it's common, but it's still important to differentiate it…

Source: Pixabay

University of Pittsburgh Joins Centers Researching Early Detection of Pancreatic Cancer

Post author:Patient Worthy Contributor
Post published:December 26, 2017
Post category:Pancreatic Cancer

Pancreatic cancer is rare, and often, patients don't show symptoms until they're already in the later stages. Top pancreatic cancer centers in the country have been working together to find…

Source: Pixabay

Cannabidiol Oral Solution Granted Fast Track Application for Prader-Willi Syndrome

Post author:Patient Worthy Contributor
Post published:December 26, 2017
Post category:Prader-Willi Syndrome

Prader-Willi syndrome is a rare condition which causes the body to feel insatiable hunger, regardless of how much a patient eats. Treatment options are currently inadequate for many patients. Today,…

Source: Pixabay

How You Can Help the Canadian Fabry Association

Post author:Patient Worthy Contributor
Post published:December 26, 2017
Post category:Fabry Disease Rare Disease

The Canadian Fabry Association wants to create awareness for rare diseases and you can help! They want to get as many people to wear a Be Rare Be You tattoo…

Source: Pixabay

Study Of Thrombotic Thrombocytopenic Purpura Medicine Demonstrates Safety

Post author:Patient Worthy Contributor
Post published:December 24, 2017
Post category:Thrombocytopenia

Caplacizumab is a medicine that has been developed to treat acquired thrombotic thrombocytopenic purpura (TTP). TTP causes serious symptoms. such as thrombocytopenia, a condition in which a patient doesn't have enough platelets. A…

Source: Pixabay

FDA Grants Orphan Drug Designation For Lennox-Gastaut Syndrome Treatment

Post author:Patient Worthy Contributor
Post published:December 24, 2017
Post category:Lennox-Gastaut syndrome

Lennox-Gastaut syndrome (LGS) is a severe and rare form of epilepsy, which causes patients to often have multiple seizures a day. It generally appears in early childhood, and is difficult…

Amy’s Acromegaly Story: Part 1

Editor’s Choice: When Sandwiches and Air Horns Change Lives

Cognitive Impairment, Testing, and Disease Burden in MPS II Discussed at Satellite Symposium

New Study Shows Mice Can Successfully Mimic Human Cancers

Satellite Symposium Discusses Long-Term Outcomes for MPS IVA and VI Patients

Editor’s Choice: Songs, Viruses, and Legal Battles

Editor’s Choice: Just Keep Swimming

Editor’s Choice: Treatment Wishes Really Do Come True

Braving the Storm: How Music and Family Helped Tara Get Through a Tough, Rare, Little-Known Disease

Editor’s Choice: When it Comes to Rare Diseases, Unity is Strength

How to Survive and Thrive: Tips From an LGS Mother

Here’s How You Can Help the Charcot-Marie-Tooth Association!

Retrophin Gift to Establish Rare Disease Network at Children’s Hospital

Editor’s Choice: Ringing in the 2018 with Awareness

Orphan Drug Designation Granted for New IPF Treatment

Editor’s Choice: New Year, New Treatments

Disappointing News for Uveitis Treatment

How Gut Bacteria Could Change Your Response to Renal Cell Carcinoma Treatment

New Trial for sGC Stimulator Treatment for Sickle Cell Disease

The Monoclonal Antibodies That Could Treat Hepatocellular Carcinoma

University of Pittsburgh Joins Centers Researching Early Detection of Pancreatic Cancer

Cannabidiol Oral Solution Granted Fast Track Application for Prader-Willi Syndrome

How You Can Help the Canadian Fabry Association

Study Of Thrombotic Thrombocytopenic Purpura Medicine Demonstrates Safety

FDA Grants Orphan Drug Designation For Lennox-Gastaut Syndrome Treatment

Let’s Work Together!

Keep Up to Date

Learn More