The beloved singer Roberta Flack has announced through a spokesperson that she is battling ALS. The brain disease called amyotrophic lateral sclerosis, or popularly known as Lou Gehrig's disease, has made it "impossible to sing and not easy to speak," the spokesperson said in a press release on Monday.
Besides the regulatory discussion, there are questions about pricing and reimbursement. Amylyx expects Relyvrio to become commercially available in the U.S. within the next four to six weeks.
Today, NPR's neuroscience correspondent Jon Hamilton checks in with Emily about this tough call for regulators: how do you weigh promising but limited evidence – and the pleas of patients desperate for hope – against the backdrop of a 100% fatal disease with hardly any other treatment options?
On September 29, 2022, the FDA made the decision to approve AMX0035, Amylyx Pharmaceuticals’ coformulation of sodium phenylbutyrate/taurursodiol, representing a monumental victory for the amyotrophic lateral sclerosis (ALS) community and standing as a testament to the continued perseverance to advance the treatment landscape.
New target discovered and delivered into the clinic in just four years using CONVERGETM, Verge’s all-in-human, AI-powered platform
VRG50635 is one of the first drugs entirely discovered and developed from a tech-enabled platform to enter clinical trials
ALS Association leaders and advocates from the ALS community will press lawmakers in Maryland to pass legislation that would prevent insurance companies from using genetic testing information to deny coverage or influence price considerations.