FDA Approves the First-Ever Treatment for Alexander Disease
Alexander disease affects perhaps one in a million people and had nothing. Zanvastro turns down production of the protein that causes it — and slowed the loss of walking speed.
The T cells that worsen tau-driven brain damage are activated outside the brain entirely. That is inconvenient for the theory and extremely convenient for drug developers.
Alexander disease affects perhaps one in a million people and had nothing. Zanvastro turns down production of the protein that causes it — and slowed the loss of walking speed.
Humana and CVS backing a cancer-navigation company at a $2B+ valuation is not really a bet on software. It is a bet that guiding patients through oncology is cheaper than not doing so.
Pulmonary hypertension from left heart disease is common, dangerous and has no approved drug therapy. A catheter that burns nerves in the pulmonary artery halved clinical worsening — in an unblinded trial.
A genome-wide CRISPR screen found a single gene whose removal turns human pancreatic duct cells into insulin producers. They lowered blood sugar in diabetic mice — while making far less insulin than the real thing.
The FDA demanded a sham brain surgery trial, then reversed itself after its leadership resigned. uniQure has now filed three-year data on a gene therapy for a disease that has defeated everything.
Weight loss reverses the number on the scale quickly. A decade-long study found the epigenetic marks obesity leaves on T cells persist five to ten years — which may explain why the risk does not fall as fast as the weight.
Celiac disease has one treatment: never eat gluten again, and hope you never do so by accident. Teva’s anti-IL-15 antibody reduced gluten-induced intestinal damage in a Phase 2a trial.
Professional societies issued their own respiratory-season vaccine guidance, developed independently of the federal advisory process. The clinical advice is unremarkable; the fact that they had to issue it is not.
Roche committed up to $1.53 billion for an antibody that has never been in a human. The price reflects how convinced pharma has become that deep B-cell depletion can reset autoimmune disease.
Takotsubo looks exactly like a heart attack until a catheter shows clear arteries. A blood-based score claims to separate the two at presentation — which is useful only if it never misses a real infarction.
Every flu vaccine on the market aims at one surface protein. GSK’s mRNA candidate adds a second — the one associated with reduced severity and transmission rather than with blocking infection.