Panelists discuss how spinal muscular atrophy is an autosomal recessive genetic disease affecting motor neurons with 3 ...

Taevion Norris’ graduation took place in Lurie Children’s Hospital because he was too sick to attend the ceremony. The hospital and staff went the whole nine yards. As “Pomp and Circumstance” played, ...

Shad’s R&R Canadian aftermarket fundraiser exceeded its stretch goal of $6 million dollars in cumulative funds raised, a ...

Panelists discuss how recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying ...

Liver damage is a well-recognised complication of gene therapies based on adeno-associated virus (AAV) vectors, and other ...

Medcare’s approach goes beyond clinical excellence. Patients benefit from a multidisciplinary team that includes experts in ...

BASEL, Switzerland I June 15, 2025 I Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today new dosing restrictions, effective immediately, for ELEVIDYS™ ...

After a thorough clinical review, the benefit-risk for the use of Elevidys in non-ambulatory patients with Duchenne has been ...

This week, a 16-year-old Toronto boy, whose bike accident left him with limited movement, will become the youngest in Ontario ...

Rgenta Therapeutics Inc. has synthesized new 2,3-dihydropyrollopyridine carboxamide compounds acting as PMS1 protein homolog 1 splicing modulators. They are reported to be useful for the treatment of ...

For patients diagnosed with ALS and other neuromuscular conditions, specialized care is critical, but often out of reach. That’s changing in the Central Valley, ...