FDA Accepts Priority Review NDA for Potential Niemann-Pick Disease Type C Drug
source: pixabay.com

FDA Accepts Priority Review NDA for Potential Niemann-Pick Disease Type C Drug

According to a story from globenewswire.com, the US Food and Drug Administration (FDA) recently accepted the New Drug Application (NDA) for arimoclomol. This application is for the use of the…

Continue Reading FDA Accepts Priority Review NDA for Potential Niemann-Pick Disease Type C Drug
Rare Disease Advocacy Group has Innovative Idea to Raise Awareness Amidst Social Distancing
source: pixabay.com

Rare Disease Advocacy Group has Innovative Idea to Raise Awareness Amidst Social Distancing

Gaucher Disease Awareness Month takes place every October, but amidst this new global pandemic, planning has taken place early to figure out how to make the month as successful as…

Continue Reading Rare Disease Advocacy Group has Innovative Idea to Raise Awareness Amidst Social Distancing
New Drug Application for Niemann-Pick Disease Type C Treatment Submitted Under Rolling Review
source: pixabay.com

New Drug Application for Niemann-Pick Disease Type C Treatment Submitted Under Rolling Review

According to a story from globenewswire.com, the biopharmaceutical company Orphazyme A/S has recently announced that it has submitted its New Drug Application (NDA) for its experimental treatment arimoclomol to the…

Continue Reading New Drug Application for Niemann-Pick Disease Type C Treatment Submitted Under Rolling Review
Arimoclomol Receives the Fast Track Designation for the Treatment of ALS
source: pixabay.com

Arimoclomol Receives the Fast Track Designation for the Treatment of ALS

CytRx Corporation has recently received the Fast Track designation for their ALS treatment, arimoclomol. This status was also given for the treatment of Niemann-Pick disease and sporadic Inclusion Body Myositis…

Continue Reading Arimoclomol Receives the Fast Track Designation for the Treatment of ALS

Sanofi is Making Strides in Lysosomal Storage Disease Research

Sanofi There are an estimated 350 million rare disease patients across the world. Many conditions still don't have treatments. However, there are scientists dedicating their lives solely to this endeavor.…

Continue Reading Sanofi is Making Strides in Lysosomal Storage Disease Research
A Primer on Clinical Trials for Patients With Gaucher Disease and Other Rare Illnesses
mcmurryjulie / Pixabay

A Primer on Clinical Trials for Patients With Gaucher Disease and Other Rare Illnesses

According to a post from gaucherdisease.org, clinical trials are an essential component to the development and testing of new therapies for Gaucher disease and other rare diseases. For some rare…

Continue Reading A Primer on Clinical Trials for Patients With Gaucher Disease and Other Rare Illnesses
India Turns to Enzyme Replacement Therapy as it Seeks to Address Rare Diseases
klbz / Pixabay

India Turns to Enzyme Replacement Therapy as it Seeks to Address Rare Diseases

According to a story from health.economictimes.indiatimes.com, the country of India is currently beginning to take more steps in order to address the dire unmet medical need of its rare disease…

Continue Reading India Turns to Enzyme Replacement Therapy as it Seeks to Address Rare Diseases