Novartis scores FDA nod for game-changing SMA treatment

Itvisma is a one-time, fixed-dose therapy designed to tackle the genetic root cause of SMA

EU clears Bristol Myers Squibb’s CAR-T therapy Breyanzi for tough-to-treat Lymphoma

The European Commission’s green light marks Breyanzi’s fourth indication in Europe and extends access to patients across all EU and EEA countries

FDA investigates fatal case linked to rare blood disorder drug Adzynma

Adzynma, approved for both adults and children with cTTP, is a recombinant enzyme therapy designed to replace the deficient ADAMTS13 enzyme

Chiesi inks agreement with AbbVie subsidiary to push next-generation brain-targeting therapies for rare diseases

Time Medical launches India’s first low-field TOF MRI for nationwide aneurysm screening

India presents one of the world’s largest untapped opportunities for neurovascular screening

Transition Bio and Voyager Therapeutics forge strategic collaboration to target ALS, FTD

The partnership aims to develop novel, selective small molecules targeting TDP-43 pathology

MIRA Pharmaceuticals advances oral Ketamir-2 into Phase 1 MAD study for chemotherapy-induced neuropathic pain

Advancing toward Phase 2a evaluation in chemotherapy-related pain, a condition with no approved treatments and potential for FDA Fast Track designation

Roche’s Elecsys pTau181 becomes first FDA-cleared blood test to rule out Alzheimer’s pathology in primary care

New test expands access to early Alzheimer’s assessment, streamlining diagnostic pathways and improving referral efficiency

Hesperos and Bayer Consumer Health develop frst human-on-a-chip model of stress-induced cognitive dysfunction

New study presents a human-relevant stress model for assessing potential therapeutics

FDA responds to evidence of possible association between autism and acetaminophen use during pregnancy

Agency initiates safety label change and notifies physicians of possible link