“I Could Die:” Vocalist from The Script Talks About Wilson’s Disease Diagnosis
Free-Photos / Pixabay

“I Could Die:” Vocalist from The Script Talks About Wilson’s Disease Diagnosis

According to a story from irishmirror.ie, Danny O'Donoghue, the vocalist and keyboard player for the Irish rock band The Script, recently talked publicly about his struggle with Wilson's disease, a…

Continue Reading “I Could Die:” Vocalist from The Script Talks About Wilson’s Disease Diagnosis
Results of Phase 3 Trial Suggest The Drug Cosentyx Could be Effective for Axial Spondyloarthritis
source: pixabay.com

Results of Phase 3 Trial Suggest The Drug Cosentyx Could be Effective for Axial Spondyloarthritis

According to a story from Ankylosing Spondylitis News, findings from a phase 3 clinical trial suggest that the drug secukinumab (marketed as Cosentyx) could be an effective treatment for non-radiographic…

Continue Reading Results of Phase 3 Trial Suggest The Drug Cosentyx Could be Effective for Axial Spondyloarthritis
This Company Just Provided a Grant for a Niemann-Pick Disease Type C Screening Program
Pexels / Pixabay

This Company Just Provided a Grant for a Niemann-Pick Disease Type C Screening Program

According to a story from ir.stockpr.com, the biotechnology company Cyclo Therapeutics, Inc. (previously known as CTD Holdings) has recently thrown its support behind a Niemann-Pick Disease Type C newborn screening…

Continue Reading This Company Just Provided a Grant for a Niemann-Pick Disease Type C Screening Program

A Teenager was Killed by a Disease so Rare it Didn’t Have a Name, But His Story Could Change the Lives of Others Like Him

According to a story from NBC News, Mitchell Herndon, a 19 year old from Missouri, recently passed away due to the progression of a rare genetic disease that is so…

Continue Reading A Teenager was Killed by a Disease so Rare it Didn’t Have a Name, But His Story Could Change the Lives of Others Like Him
Enrollment Begins for Amyotrophic Lateral Sclerosis Clinical Trial
DarkoStojanovic / Pixabay

Enrollment Begins for Amyotrophic Lateral Sclerosis Clinical Trial

According to a story from BioPortfolio, the biopharmaceutical company Collaborative Medicinal Development, LLC has recently announced that the first patient has been enrolled in a clinical trial that will test…

Continue Reading Enrollment Begins for Amyotrophic Lateral Sclerosis Clinical Trial

Study Reveals a More Efficient Approach for Diagnosing Certain Cases of Hereditary Angioedema

According to a story from Angioedema News, a recent study discusses a genetic procedure called allelic discrimination as a method for diagnosing hereditary angioedema. The study concluded that this method…

Continue Reading Study Reveals a More Efficient Approach for Diagnosing Certain Cases of Hereditary Angioedema
Experimental Non-Steroid Treatment Displays Potential for Congenital Adrenal Hyperplasia
Anqa / Pixabay

Experimental Non-Steroid Treatment Displays Potential for Congenital Adrenal Hyperplasia

According to a story from Healio, the results from a recent phase 2A clinical trial have demonstrated the effectiveness of an experimental drug in treating congenital adrenal hyperplasia (CAH). The…

Continue Reading Experimental Non-Steroid Treatment Displays Potential for Congenital Adrenal Hyperplasia
A Girl Diagnosed with Batten Disease got Treated with a Drug Developed Specifically for Her
DarkoStojanovic / Pixabay

A Girl Diagnosed with Batten Disease got Treated with a Drug Developed Specifically for Her

According to a story from Technology Review, the story of Mila Makovec shows the potential impact that personalized medicine could have on patients while also revealing some of its limitations.…

Continue Reading A Girl Diagnosed with Batten Disease got Treated with a Drug Developed Specifically for Her
Data Indicates That Gene Therapy for Spinal Muscular Atrophy Could be Useful for Older Patients
madartzgraphics / Pixabay

Data Indicates That Gene Therapy for Spinal Muscular Atrophy Could be Useful for Older Patients

According to a story from MedCity News, interim data from a phase 1/2 clinical trial testing Zolgensma, a gene therapy for spinal muscular atrophy, in patients between the ages of…

Continue Reading Data Indicates That Gene Therapy for Spinal Muscular Atrophy Could be Useful for Older Patients
Two Nonprofits Award $200,000 Grant for Development of Duchenne Muscular Dystrophy Patient Reported Outcomes
source: pixabay.com

Two Nonprofits Award $200,000 Grant for Development of Duchenne Muscular Dystrophy Patient Reported Outcomes

According to a story from PR Newswire, the nonprofit groups Duchenne UK and Parent Project Muscular Dystrophy (PPMD) have jointly awarded grant funding to the tune of $200,000 to Dr.…

Continue Reading Two Nonprofits Award $200,000 Grant for Development of Duchenne Muscular Dystrophy Patient Reported Outcomes
Grant to Fund Critical International Amyotrophic Lateral Sclerosis Research
TBIT / Pixabay

Grant to Fund Critical International Amyotrophic Lateral Sclerosis Research

According to a story from med.miami.edu, an international research consortium known as Clinical Research in ALS and Related Disorders for Therapeutic Development (CReATe) has just been awarded a renewal grant from…

Continue Reading Grant to Fund Critical International Amyotrophic Lateral Sclerosis Research

An Experimental Cystic Fibrosis Treatment Could Make This Patient’s Greatest Wish Come True

According to a story from abc.net.au, Darren Bullock is getting pretty long in the tooth---at least for a cystic fibrosis patient. He is 44 years old, and his lungs currently…

Continue Reading An Experimental Cystic Fibrosis Treatment Could Make This Patient’s Greatest Wish Come True