ICYMI: The Affordable Care Act May be Ruled Unconstitutional: Patient Groups Unite to Ask for a Prompt Decision
source: pixabay.com

ICYMI: The Affordable Care Act May be Ruled Unconstitutional: Patient Groups Unite to Ask for a Prompt Decision

As originally reported in NewsRoom, the US's political volatility over the past years has thrown many policies and legislation into debate. While switching the party in both the executive and…

Continue Reading ICYMI: The Affordable Care Act May be Ruled Unconstitutional: Patient Groups Unite to Ask for a Prompt Decision
How a Community has Rallied Around a Teenage Girl with Glioma
https://pixabay.com/en/children-road-distant-supportive-1149671/

How a Community has Rallied Around a Teenage Girl with Glioma

When Lily Wythe, a 14-year-old from Eastwood in England, was diagnosed with diffuse pontine glioma (also known as DIPG), her friends and family knew they had to do something to…

Continue Reading How a Community has Rallied Around a Teenage Girl with Glioma
Will Therapies for Neurological Disorders Bring Forth a New Era for Biotech?
source: pixabay.com

Will Therapies for Neurological Disorders Bring Forth a New Era for Biotech?

According to a story from BioBuzz, the innovation of a platform for the delivery of gene therapies could help trigger a golden age of development for this new class of…

Continue Reading Will Therapies for Neurological Disorders Bring Forth a New Era for Biotech?
Phase 2 Trial for Amyotrophic Lateral Sclerosis has Completed Enrollment
source: pixabay.com

Phase 2 Trial for Amyotrophic Lateral Sclerosis has Completed Enrollment

FightMND is an Australian nonprofit organization dedicated to improving the lives of motor neurone disease patients through funding research for cures. They've successfully provided millions toward research initiatives and clinical…

Continue Reading Phase 2 Trial for Amyotrophic Lateral Sclerosis has Completed Enrollment

“If You’ve Grown Up With Your Disease From Day One, You Never Know What It’s Like to be Healthy.” Patient Stories in the #IAmNumber17 Campaign

As originally reported in PR Week; pharma company Takeda has been joined a plethora of rare patients groups to create the 'I am Number 17 ' campaign which features 17…

Continue Reading “If You’ve Grown Up With Your Disease From Day One, You Never Know What It’s Like to be Healthy.” Patient Stories in the #IAmNumber17 Campaign
This Glomerulonephritis Patient has Been Receiving Dialysis Treatment for Over 30 Years. Now he Shares his Discipline with Other Patients
source: pixabay.com

This Glomerulonephritis Patient has Been Receiving Dialysis Treatment for Over 30 Years. Now he Shares his Discipline with Other Patients

  According to a recent article in the MalayMail, it occurred to Abdullah’s doctor that the 67-year-old would be an inspiration for others who have kidney failure. His nephrologist, Dr.…

Continue Reading This Glomerulonephritis Patient has Been Receiving Dialysis Treatment for Over 30 Years. Now he Shares his Discipline with Other Patients
First International Awareness Day for Alagille Syndrome was Held January 24th
source: pixabay.com

First International Awareness Day for Alagille Syndrome was Held January 24th

International Alagille Awareness Day The very first International Alagille Awareness day was held on January 24th, 2020. What is Alagille syndrome? It is a rare disease which effects the heart,…

Continue Reading First International Awareness Day for Alagille Syndrome was Held January 24th
ICYMI: The Manufacturer of Spinal Muscular Atrophy Drug Zolgensma has Made a Lottery for Life
source: pixabay.com

ICYMI: The Manufacturer of Spinal Muscular Atrophy Drug Zolgensma has Made a Lottery for Life

As originally reported in Euronews, there is limited supply of expensive medicines for rare diseases, so it can be difficult to prioritize which patients will have their lives saved when…

Continue Reading ICYMI: The Manufacturer of Spinal Muscular Atrophy Drug Zolgensma has Made a Lottery for Life
The Effects of Race on Pulmonary Arterial Hypertension Outcomes
source: pixabay.com

The Effects of Race on Pulmonary Arterial Hypertension Outcomes

According to a story from consultant360.com, a recent study was conducted in order to determine if racial ancestry had any impacts on outcomes in the rare disease pulmonary arterial hypertension…

Continue Reading The Effects of Race on Pulmonary Arterial Hypertension Outcomes
India Has Introduced a New Rare Disease Policy Draft, But it Doesn’t Benefit all Rare Patients
source: pixabay.com

India Has Introduced a New Rare Disease Policy Draft, But it Doesn’t Benefit all Rare Patients

India first drafted a national rare disease policy back in 2017. Unfortunately, it was never enacted due to budgeting and implementation issues. This draft included suggestions such as a corpus…

Continue Reading India Has Introduced a New Rare Disease Policy Draft, But it Doesn’t Benefit all Rare Patients
Seelos Therapeutics Proposes New Treatment for Sanfilippo Syndrome
source: pixabay.com

Seelos Therapeutics Proposes New Treatment for Sanfilippo Syndrome

Seelos Therapeutics has won a meeting with the European Medicines Agency (EMA) to advance its therapy, trehalose, for the treatment of neuro-degenerative diseases, specifically Sanfilippo syndrome. At this meeting, which…

Continue Reading Seelos Therapeutics Proposes New Treatment for Sanfilippo Syndrome
A 1,000 Patient Trial for Acute Lymphoblastic Leukemia and Other Hematologic Malignancies is Now Enrolling
source: pixabay.com

A 1,000 Patient Trial for Acute Lymphoblastic Leukemia and Other Hematologic Malignancies is Now Enrolling

The Platform Notable's automated technology platform was created to help predict which patients would respond better to which therapies. As no patient is the same, this study is paramount. Notable…

Continue Reading A 1,000 Patient Trial for Acute Lymphoblastic Leukemia and Other Hematologic Malignancies is Now Enrolling
As the ACA Faces Another Court Challenge, Patient Groups Urge Quick Action
source: pixabay.com

As the ACA Faces Another Court Challenge, Patient Groups Urge Quick Action

According to a story from newsroom.heart.org, one of the most substantial domestic policy achievements of the Obama administration was the passage of the Affordable Care Act (ACA). Since its passage,…

Continue Reading As the ACA Faces Another Court Challenge, Patient Groups Urge Quick Action
Let’s Talk Spoonies and Compression Socks!
Compression socks come in all different styles and patterns so even though this image is from a stock website, we have no way of knowing these guys AREN'T wearing compression socks.

Let’s Talk Spoonies and Compression Socks!

I've been hearing people buzz about compression socks/stockings for a while, but I've never invested in a pair myself. They're basically what they sound like-- socks or stockings that... compress.…

Continue Reading Let’s Talk Spoonies and Compression Socks!
Searching for the Cause of Lung Disease in Systemic Juvenile Idiopathic Arthritis
source: pixabay.com

Searching for the Cause of Lung Disease in Systemic Juvenile Idiopathic Arthritis

  According to a recent article in MedPage Today’s 2019 year-end review, the number of life-threatening lung diseases in systemic juvenile idiopathic arthritis (sJIA) patients has been increasing. sJIA is…

Continue Reading Searching for the Cause of Lung Disease in Systemic Juvenile Idiopathic Arthritis
Rylee’s Story: A Cyclic Vomiting Syndrome Diagnosis Brings Support from the Community
source: pixabay.com

Rylee’s Story: A Cyclic Vomiting Syndrome Diagnosis Brings Support from the Community

  A correct diagnosis is often difficult to obtain, especially for a rare disease. Symptoms can be attributed to more common conditions, or they can be written off entirely. Rylee…

Continue Reading Rylee’s Story: A Cyclic Vomiting Syndrome Diagnosis Brings Support from the Community
Risdiplam Delivers An Improvement to Infants with Spinal Muscular Atrophy Type 1
source: pixabay.com

Risdiplam Delivers An Improvement to Infants with Spinal Muscular Atrophy Type 1

Genentech (Roche Group) announced positive results of its FIREFISH trial in a recent Business Wire news release.  The second part of the study evaluated risdiplam’s efficacy on infants ages one…

Continue Reading Risdiplam Delivers An Improvement to Infants with Spinal Muscular Atrophy Type 1
Close Menu