DNA Sequencing Took Years, Now it Takes Hours; As a Result, Researchers Have Discovered The Cause of Four Rare Diseases

Researchers at the University of Tokyo have discovered that one segment of DNA is responsible for four rare diseases. As reported in Science Daily, the university team also believes that…

Continue Reading DNA Sequencing Took Years, Now it Takes Hours; As a Result, Researchers Have Discovered The Cause of Four Rare Diseases

Experimental Treatment for Acute Myeloid Leukemia Earns Orphan Drug Designation

According to a story from globenewswire.com, the biopharmaceutical company Mustang Bio, Inc. has recently announced that the US Food and Drug Administration (FDA) has recently awarded the company Orphan Drug…

Continue Reading Experimental Treatment for Acute Myeloid Leukemia Earns Orphan Drug Designation
Apellis Pharmaceuticals Launches Program Investigating Potential of APL-9 for Preventing AAV Resistance in Gene Therapy
luvqs / Pixabay

Apellis Pharmaceuticals Launches Program Investigating Potential of APL-9 for Preventing AAV Resistance in Gene Therapy

According to a press release from the Kentucky-based Apellis Pharmaceuticals, the Company has initiated a program to investigate the use of APL-9, an investigational drug, in gene therapies that involve…

Continue Reading Apellis Pharmaceuticals Launches Program Investigating Potential of APL-9 for Preventing AAV Resistance in Gene Therapy
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
Dr. Rahul Desikan, Amyotrophic Lateral Sclerosis Patient and Researcher, Dies
webandi / Pixabay

Dr. Rahul Desikan, Amyotrophic Lateral Sclerosis Patient and Researcher, Dies

According to a story from rediff.com, Dr. Rahul Desikan was a committed amyotrophic lateral sclerosis researcher. Then, in a cruel twist of irony, he was diagnosed with the disease himself.…

Continue Reading Dr. Rahul Desikan, Amyotrophic Lateral Sclerosis Patient and Researcher, Dies
European Medicines Agency Grants Experimental Cystic Fibrosis Drug Orphan Drug Designation
source: pixabay.com

European Medicines Agency Grants Experimental Cystic Fibrosis Drug Orphan Drug Designation

According to a press release from Aridis Pharmaceuticals, the Company has received Orphan Drug designation from the European Medicines Agency for its experimental cystic fibrosis-related lung infection treatment AR-501. The…

Continue Reading European Medicines Agency Grants Experimental Cystic Fibrosis Drug Orphan Drug Designation
Close Menu