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Phase 2 Friedreich’s Ataxia Trial Given Green Light in Spain

Phase 2 Friedreich’s Ataxia Trial Given Green Light in Spain

According to a story from BioSpace, the biotechnology company Minoryx Therapeutics recently announced that the company has received official approval from the Spanish Agency of Medicines and Medical Devices (AEMPS)…

Continue Reading Phase 2 Friedreich’s Ataxia Trial Given Green Light in Spain
First Patient Enrolled in Phase 2 Trial for Experimental RRMS Drug

First Patient Enrolled in Phase 2 Trial for Experimental RRMS Drug

According to a publication from Multiple Sclerosis News Today, German biotechnology company Immunic Therapeutics has successfully enrolled the first participant for its phase 2 clinical trial for experimental relapsing-remitting multiple…

Continue Reading First Patient Enrolled in Phase 2 Trial for Experimental RRMS Drug
A New Drug With the Potential to Treat Hypothalamic Obesity

A New Drug With the Potential to Treat Hypothalamic Obesity

According to a recent article in the Globe, the Danish biotech company Saniona AB has initiated a Phase 2a clinical trial of Tesomet for the treatment of hypothalamic obesity, a rare…

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Phase 2 Clinical Trial for Pulmonary Hypertension has Officially Begun

Phase 2 Clinical Trial for Pulmonary Hypertension has Officially Begun

About Pulmonary Hypertension Pulmonary Hypertension (PH) is a form of high blood pressure which affects the arteries. Primary Pulmonary Hypertension refers to a type of the condition whose origin is unknown. Secondary…

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Positive Results from Phase 2 Trial Could Lead to Lower Doses of Corticosteroids for Pemphigus Patients

Positive Results from Phase 2 Trial Could Lead to Lower Doses of Corticosteroids for Pemphigus Patients

Pemphigus Pemphigus is a rare autoimmune condition which causes the skin to blister. There are two main forms of the disease called pemphigus foliaceus (PF) and pemphigus vulgaris (PV). Pemphigus…

Continue Reading Positive Results from Phase 2 Trial Could Lead to Lower Doses of Corticosteroids for Pemphigus Patients
Adverum Releases Preclinical Data of Experimental Wet AMD Drug

Adverum Releases Preclinical Data of Experimental Wet AMD Drug

A press release put out last month by American pharmaceutical company Adverum Biotechnologies announced some of the long-term preclinical safety data collected about their experimental drug ADVM-022. The drug hopes…

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Population Genomics: Sixth Annual Rare Disease Genomics Symposium

Population Genomics: Sixth Annual Rare Disease Genomics Symposium

Patient Worthy has the distinct pleasure of attending the Sixth Annual Rare Disease Genomics Symposium at the University of Alabama at Birmingham (UAB) this weekend. Today, we heard from lead…

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Alexion’s Ultomiris™ Met the Primary Objective in a Phase 3 Trial of Atypical Hemolytic Uremic Syndrome Patients

Alexion’s Ultomiris™ Met the Primary Objective in a Phase 3 Trial of Atypical Hemolytic Uremic Syndrome Patients

Alexion Pharmaceuticals, Inc., a company that is advancing a rare disease pipeline in complement biology, recently issued a series of press releases announcing positive results of a Phase 3 study…

Continue Reading Alexion’s Ultomiris™ Met the Primary Objective in a Phase 3 Trial of Atypical Hemolytic Uremic Syndrome Patients
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