The White House Is Supporting Right to Try Law
A law that would give terminally ill patients easier access to medicines that are not yet FDA approved has the support of President Donald Trump, according to The Wall Street Journal. Supporters of the bill also met with Vice President Mike Pence. Trump has asserted that the many FDA rules cause unnecessary barriers to drugs getting approved. The FDA has a compassionate use program for people with terminal illnesses, but proponents of the Right to Try law contend that FDA rules get in the way of compassionate access.
Paul Ryan: ACA Replacement by End of Year
There has been a lot of uncertainty surrounding the current status of repealing and replacing the Affordable Care Act (ACA). According to Reuters, House Speaker Paul Ryan, R-Wisconsin, has said a replacement for the healthcare law would be completed by the end of 2017. This announcement comes on the heels of Trump saying in an interview that replacing the ACA may take until next year. Getting Representative Tom Price, R-Georgia, confirmed as HHS secretary is an important step toward replacing the ACA, Ryan said.
Organizations Express Support for Vaccine Safety
More than 350 organizations have sent a letter expressing their support for the safety of vaccines to the president, who has embraced claims that discredit vaccine safety and has met with anti-vaccine groups. The Washington Post reported that the letter outlines the benefits of vaccines and states that claims vaccines are unsafe have been disproven in the medical literature. The 350 organizations that signed the letter include medical, advocacy, and professional organizations, including the American Medical Association, the Children’s Defense Fund, and the March of Dimes.
Neurologists Share Tips for Securing Patient Access to Gene Therapies
March 19th 2025Tenacious efforts at every level, from the individual clinician to the hospital to the state to Congress, will be needed to make sure patients can access life-saving gene therapies for neuromuscular diseases.
Read More
EMBARK Data Show Continued Improvements With DMD Gene Therapy
March 19th 2025Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that benefits in functional outcomes, gene expression, and muscle imaging persist 2 years after receiving the gene therapy.
Read More