The FDA’s proposed Rare Disease Evidence Principles review process is a starting point for getting rare disease therapies across the finish line, but industry leaders say there are more concrete steps the regulator could take to help patients.
Rare Disease Experts See Futility in FDA’s Proposed Framework Read More »
A decade-long journey has come to an end for Stealth BioTherapeutics and the Barth syndrome community with the first-ever treatment for this uncommon mitochondrial disease. CEO Reenie McCarthy called it a “pivotal victory” that “offers hope for expedited regulatory attention to other ultra-rare diseases.”
Stealth Wins Expedited FDA Nod for First-Ever Barth Syndrome Treatment Read More »
The payment resolves allegations brought by the Department of Justice that Exactech violated the False Claims Act by selling knee implants with components that failed prematurely.
Exactech agrees to $8M settlement over defective knee implants Read More »
After a tension-packed two days that saw changes to the MMRV vaccine schedule and COVID-19 recommendations, as well as a delayed hepatitis B vaccine vote, policy experts expressed concern with the reconstituted committee’s dearth of previous experience and “understanding” of their role.
ACIP Day 2 Reveals Confusion, ‘Lack of Knowledge,’ on Revamped Committee Read More »
Fuente: Freepik Trabajar con niños es una vocación que despierta ilusión y muchas ganas de aprender cada día. Para quienes sienten esa llamada, la primera opción que suele venir a la cabeza es estudiar magisterio, aunque no siempre es el camino q …
Trabajar con niños sin estudiar magisterio: cursos que te abren camino Read More »
The policy was originally proposed by the Biden administration in November, but comes amid a larger push for more transparency around payers’ provider networks from health officials in the Trump regime.
Medicare Advantage plans to share provider directories under CMS final rule Read More »
Regulation of lab developed tests returns to its status before the agency attempted to expand its jurisdiction and industry groups sued to stop it.
The return to profitability is a victory for the system, which reported a $1 billion loss last year as it struggled to recover from a cyberattack.
Members of ACIP postponed a planned vote on withholding some infants’ first hepatitis B vaccine dose, adding more confusion to an already muddied meeting.
CDC advisers postpone vote to delay some newborns’ hepatitis B shot Read More »
The purchase gives Biogen full access to an implantable device that could help patients avoid the repeat lumbar punctures required for drugs like Spinraza.
Biogen to buy startup Alcyone, eyeing easier delivery of RNA drugs Read More »
