New drugs in development or already approved by the FDA are so complex that additional training is required. New gene-based remedies for rare conditions such as Angelman syndrome and inherited retinal disease (IRD) necessitate specialized training for physicians. If new medical discoveries are to have an impact, physicians must take the time to comprehend their nuances.
As per Healthnews, Foundation for Angelman Syndrome Therapeutics, or FAST for short, is one of the pioneers in Angelman syndrome research. Angelman syndrome is a genetically complex disorder of the nervous system. Angelman syndrome is characterized by delayed development, intellectual disability, severe speech impairment, and difficulty moving and sustaining balance. According to FAST, one in 15,000 persons has Angelman syndrome.
The mission of FAST is to aid those with Angelman syndrome and to conduct research into therapies and treatments for the disease. According to FAST’s website, 88% of every dollar donated is used to advance research. GeneTx Biotherapeutics, a subsidiary of FAST, is developing antisense oligonucleotides (ASOs) as a prospective Angelman syndrome treatment.
Antisense oligonucleotides are minuscule strands of genetic material synthesized in a laboratory that bind to messenger RNA molecules in our cells that are responsible for protein synthesis. Antisense oligonucleotides bind to their target RNA to prevent the RNA from producing a protein or to cause our cells to degrade the RNA. In December, FAST donated $5 million to Rush University Medical Center in Chicago, Illinois, for Angelman syndrome research and training for medical professionals.
Elizabeth Berry-Kravis, M.D., Ph.D., is the director of the Rush Pediatric Neurosciences F.A.S.T Center for Translational Research, and she states, “There are few centers that investigate or treat rare neurological disorders in the United States. By bringing educational training programs to support these objectives, the aim is to assist centers in meeting the growing demand for early-stage clinical trials for children with these disorders.
One of the treatments in development is Ultragenyx Pharmaceutical Inc.’s GTX-102. The medication is an antisense oligonucleotide intended to inhibit the expression of UBE3A-ATS. The UBE3A gene helps produce proteins that degrade other intracellular proteins. Angelman syndrome can result from mutations in the UBE3A gene.
According to a recent Wall Street Journal report, Ultragenyx is having trouble locating institutions with sufficiently knowledgeable physicians to initiate or administer GTX-102. These remedies seem to be intended for the future, not the present.
Training for new, futuristic medicines is not on the agenda for all physicians. Doctors who wish to register with the Drug Enforcement Administration (DEA) after June 27, 2023, must complete eight hours of coursework on the treatment of opioid-dependent patients. The new modification is a result of the December passage of the Consolidated Appropriations Act.
The Providers Clinical Support System has developed a 23-module training curriculum to assist those in need of education. The new training is motivated in part by the ongoing opioid pandemic in the United States. The CDC estimates that the number of opioid-related drug overdose fatalities will increase from 70,029 in 2020 to 80,821 in 2021.
