A Proof-of-Concept Study for an Experimental Hurler Syndrome Treatment is Going Well so Far
sasint / Pixabay

A Proof-of-Concept Study for an Experimental Hurler Syndrome Treatment is Going Well so Far

According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Orchard Therapeutics recently announced the release of some data from an ongoing proof-of-concept study. This special trial is testing the company's…

Continue Reading A Proof-of-Concept Study for an Experimental Hurler Syndrome Treatment is Going Well so Far

CRISPR Gene Editing Promises to Cure 10,000 diseases. Now in Its First Human Clinical Trial. Will it Eventually Deliver?

  Clustered Regularly Interspaced Short Palindromic Repeats is more commonly known as CRISPR. As reported recently in Science News, the CRISPR/Cas9 “molecular scissors” is slated to make its highly anticipated debut…

Continue Reading CRISPR Gene Editing Promises to Cure 10,000 diseases. Now in Its First Human Clinical Trial. Will it Eventually Deliver?
FDA Begins Review of Biologics License Application for Neuromyelitis Optica Spectrum Disorder Drug
mohamed_hassan / Pixabay

FDA Begins Review of Biologics License Application for Neuromyelitis Optica Spectrum Disorder Drug

According to a press release from Maryland-based Viela Bio, the US Food and Drug Administration (FDA) recently agreed to review the Company's Biologics License Application for its neuromyelitis optica spectrum…

Continue Reading FDA Begins Review of Biologics License Application for Neuromyelitis Optica Spectrum Disorder Drug
A Tale of Two Sisters With Cystic Fibrosis Highlights the Barriers to Treatment Access That Still Remain
Free-Photos / Pixabay

A Tale of Two Sisters With Cystic Fibrosis Highlights the Barriers to Treatment Access That Still Remain

According to a story from the Sunday Post, sisters Shona and Kirsty Young, aged 24 and 29 years respectively, were both born with the devastating rare disease cystic fibrosis. However,…

Continue Reading A Tale of Two Sisters With Cystic Fibrosis Highlights the Barriers to Treatment Access That Still Remain

Zogenix Acquires Modis in $400 Million Buyout, Adds Experimental TK2 mDNA Depletion Syndrome Treatment to Pipeline

According to a press release from California-based biotechnology company Zogenix, the Company recently completed a multi-million dollar acquisition of Modis Therapeutics. Before its acquisition, Modis focused on the development of…

Continue Reading Zogenix Acquires Modis in $400 Million Buyout, Adds Experimental TK2 mDNA Depletion Syndrome Treatment to Pipeline
Korean Company Predicts Your Risk of Developing Certain Genetic Conditions by Sequencing Your Genome
Source: Pixabay

Korean Company Predicts Your Risk of Developing Certain Genetic Conditions by Sequencing Your Genome

According to a publication from the Korea Times, the Eone Diagnomics Genome Center in Incheon, South Korea, is working to improve patients' accessibility to their own genetic sequence. Researchers believe…

Continue Reading Korean Company Predicts Your Risk of Developing Certain Genetic Conditions by Sequencing Your Genome
A Recent Discovery Could Make Cancer Treatments More Effective
ColiN00B / Pixabay

A Recent Discovery Could Make Cancer Treatments More Effective

According to a story from news-medical.net, scientists may have made a significant breakthrough discovery that could make cancer treatment more effective. The discovery involves a new method that breaks through…

Continue Reading A Recent Discovery Could Make Cancer Treatments More Effective

FDA Begins Priority Review of Xtandi, a Drug Candidate for Metastatic Hormone-Sensitive Prostate Cancer

According to a press release from American pharmaceutical giant Pfizer, Inc. the FDA recently agreed to review a supplemental New Drug Application for Xtandi. Xtandi was first approved for the…

Continue Reading FDA Begins Priority Review of Xtandi, a Drug Candidate for Metastatic Hormone-Sensitive Prostate Cancer
FDA Grants Regenerative Medicine Advanced Therapy Designation to Investigational X-Linked SCID Therapy
rawpixel / Pixabay

FDA Grants Regenerative Medicine Advanced Therapy Designation to Investigational X-Linked SCID Therapy

According to a press release from American biotechnology company Fortress Biotech, the American Food and Drug Administration (FDA) recently awarded Regenerative Medicine Advanced Therapy designation to the experimental gene therapy…

Continue Reading FDA Grants Regenerative Medicine Advanced Therapy Designation to Investigational X-Linked SCID Therapy
Beta Blocker Fails to Stop Hereditary Hemorrhagic Telangiectasia-Associated Nosebleeds
jarmoluk / Pixabay

Beta Blocker Fails to Stop Hereditary Hemorrhagic Telangiectasia-Associated Nosebleeds

According to a recent study published in Nature Research, a team of scientists studied the efficacy of timolol in treating nosebleeds associated with hereditary hemorrhagic telangiectasia. Timolol is a beta…

Continue Reading Beta Blocker Fails to Stop Hereditary Hemorrhagic Telangiectasia-Associated Nosebleeds

ICYMI: An Experimental Treatment for POMC and LEPR Deficiency Obesity Appears Effective

According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Rhythm Pharmaceuticals, Inc., has recently announced the results from two phase 3 clinical trials. These trials were testing the company's experimental…

Continue Reading ICYMI: An Experimental Treatment for POMC and LEPR Deficiency Obesity Appears Effective
CPRIT Awards Emtora Biosciences $3 Million Grant to Develop Investigational Familial Adenomatous Polyposis Drug
source: pixabay.com

CPRIT Awards Emtora Biosciences $3 Million Grant to Develop Investigational Familial Adenomatous Polyposis Drug

According to a recent press release, Texas-based biotechnology company Emtora Biosciences was chosen earlier this month to receive a $3 million grant from the Cancer Prevention and Research Institute of…

Continue Reading CPRIT Awards Emtora Biosciences $3 Million Grant to Develop Investigational Familial Adenomatous Polyposis Drug
Experimental Treatment for Pancreatic Cancer has Earned Orphan Drug Designation
source: pixabay.com

Experimental Treatment for Pancreatic Cancer has Earned Orphan Drug Designation

According to a story from BioSpace, the biopharmaceutical company CrystalGenomics, Inc. recently announced that the US Food and Drug Administration (FDA) has granted the company's investigational drug candidate CG-745 Orphan…

Continue Reading Experimental Treatment for Pancreatic Cancer has Earned Orphan Drug Designation

Expectations For This Clinical Trial Were to Slow Down ALS Progression; Instead it Reversed One Man’s Symptoms

  According to a recent article published in News NWA, in 2017 Mark Bedwell, of Fort Smith, Arkansas had been told that based upon the rapid progression of his amyotrophic…

Continue Reading Expectations For This Clinical Trial Were to Slow Down ALS Progression; Instead it Reversed One Man’s Symptoms
ICYMI: With the FDA’s Third Approval for Tissue Agnostic Therapies, Oncologists are Headed to the Next Level in Cancer Treatment
source: pixabay.com

ICYMI: With the FDA’s Third Approval for Tissue Agnostic Therapies, Oncologists are Headed to the Next Level in Cancer Treatment

Since the 1940s oncologists have been focusing on tumors at the location in the body where the tumors originated. According to a recent FDA press release, this is the third…

Continue Reading ICYMI: With the FDA’s Third Approval for Tissue Agnostic Therapies, Oncologists are Headed to the Next Level in Cancer Treatment