Rare Diseases
FDA/CDC
ALS drug gets FDA panel thumbs-up after rare second look
FDA officials in the meeting stressed the importance of considering unmet medical need in ALS in the panel’s decision-making process.
From the Journals
Ketamine promising for rare condition linked to autism
Using parent-report instruments to assess treatment effects, ketamine was associated with “nominally significant” improvement in a variety of...
News from the FDA/CDC
CDC warns of enterovirus strain linked to polio-like condition
The advisory also urged providers to “strongly consider AFM in patients with acute flaccid limb weakness, especially after respiratory illness or...
Hitting a Nerve
Polio: The unwanted sequel
Cracks in herd immunity have allowed polio to return.
Latest News
Two deaths from liver failure linked to spinal muscular atrophy drug
These are the first known fatal cases of acute liver failure associated with the drug, which the company notes was a known side effect included in...
Literature Review
First-ever Huntington staging system may jump-start drug development for early-stage disease
Researchers liken the Huntington’s disease Integrated Staging System (HD-ISS) to the system currently used to stage cancer.
FDA/CDC
FDA unveils 5-year plan for ALS and other neurodegenerative diseases
“We recognize the urgent need for new treatments that can both improve and extend the lives of people diagnosed with these diseases.”
Conference Coverage
Tofersen linked to slow, positive effects in ALS
One-year clinical trial results show a deceleration in functional decline that is similar, but “more pronounced” than the previously reported 6-...
Conference Coverage
Inebilizumab beneficial across genotypes in NMOSD
"These data illustrate how mechanistic precision in treatment design can help patients gain benefit from their regimen regardless of the genetic...
FDA/CDC
FDA expands indication for spinal muscular atrophy drug
After 12 months of risdiplam treatment, the majority of presymptomatic infants with SMA reached key motor milestones.
FDA/CDC
FDA approves oral form of ALS drug edaravone
Compared with the IV formation of the drug, the oral form has been shown to generate comparable levels of active drug in the bloodstream.