Pediatric Rare diseases and Rare Pediatric Disease Designation FDA will award priority review vouchers to sponsors of certain rare pediatric disease product applications that meet the criteria specified. Section 529 of the FD&C Act is intended to encourage development of new drug and biological products for the prevention and treatment of rare pediatric diseases. Section […]
The World Orphan Drug Congress USA will be a live virtual conference. BBCR’s Team invites you to send an email at [email protected]/bbcr2. We hope to meet many of you that will attend the World Orphan Drug Congress. We are very much interested in learning about your programs to determine if BBCR’s expertise in Rare Diseases […]
Patients with mild COVID 19 infection, from a variety of age groups, were still experiencing major symptoms for months after COVID 19 disease is over. Read full article: https://jamanetwork.com/journals/jama/fullarticle/2768351 The Jama study was conducted in 143 COVID 19 patients. The mean age was 56.5 (SD, 14.6) years (range, 19-84 years), and 53 (37%) were women. […]
A transient neutralizing Ab (nAb) response is a feature shared by both COVID19 infection and the coronaviruses that cause common colds (https://www.medrxiv.org/content/10.1101/2020.07.09.20148429v1) Antibody (Ab) responses to SARS-CoV-2 can be detected in most infected individuals 10-15 days following the onset of COVID-19 symptoms. Seroconversion occurred in >95% of cases and neutralizing antibody (nAb) responses. All subjects, […]
SARS-CoV-2 outbreaks that cannot be explained by large droplets and contact with surfaces alone. Droplets from speech can float in air for eight minutes. A growing body of evidence suggests beyond any reasonable doubt that the virus spreads indoors through tiny aerosols—a finding that should be reflected in the WHO’s recommendations. Like the flu, COVID-19 […]
Scientists launched studies in search of genes that could explain why some people infected with SARS-CoV-2 get really sick, while others have only mild symptoms. We know that chronic health conditions—such as hypertension and diabetes can play a role, but there are also evidences that people’s genes can influence how their bodies react to other […]
Techniques that bypass or trick the guardian of the central nervous system. Developing drugs that can penetrate into the brain requires some creativity. The approaches vary, each technique comes with its benefits and drawbacks, making it appropriate for some patients or drug types but not others. In some diseases, you might want the drug everywhere; […]
Preliminary data suggest that survivors of SARS-CoV-2 infection may be susceptible to reinfection within weeks or months. One study in ~1,500 COVID patients show that 10 percent % of these patients had undetectable antibody levels within weeks of first symptoms or lost their antibodies two to three months after recovering from the infection. Early works […]
Lesions in the lungs of patients with pneumonia caused by a SARS-CoV-2 infection are distinct from those caused by bacteria. SARS-CoV-2 is known to damage lung tissue as many COVID-19 patients develop pneumonia, which can progress to respiratory failure and sometimes death. In chest CT scans lungs of COVID-19 patients show cloudy lesion patterns that […]
People tend to believe that a repurposed therapy can never be truly novel or transformative. Nothing could be further from the truth. One attractive option of Drug Repurposing is to use a scientific approach to identify new uses for existing drugs. About a third of orphan approvals by the FDA since the program began have […]