Can Servier’s $2.65bn Edgewise deal reshape the muscular dystrophy drug race?
Servier is buying sevasemten before pivotal muscular dystrophy data. The deal gives Edgewise cash and investors a sharper cardiology story.
Angelini Pharma bets $4.1bn on rare neurology scale with Catalyst Pharmaceuticals
Angelini gains U.S. rare disease scale. Catalyst gets certainty, but Firdapse concentration keeps the real test ahead.
Could Entos Pharmaceuticals’ Fusogenix platform open a real path to treatment in L-CMD?
Entos Pharmaceuticals and the L-CMD Research Foundation are targeting a curative L-CMD therapy. Read what this could change for rare disease treatment.
Sarepta’s sirolimus gambit: Can enhanced immunosuppression rescue ELEVIDYS for non-ambulatory Duchenne patients?
Sarepta’s ELEVIDYS Cohort 8 targets non-ambulatory Duchenne patients with a sirolimus regimen. What the trial design reveals about the path to label restoration.
What the FDA decision on PTC Therapeutics’ Translarna reveals about Duchenne muscular dystrophy drug approvals
PTC Therapeutics has withdrawn its U.S. filing for Translarna. Find out what this means for Duchenne drug development and FDA expectations.
Can Sarepta’s ELEVIDYS redefine Duchenne therapy? Inside the 3-year EMBARK data
Sarepta’s gene therapy ELEVIDYS shows 3-year durability in Duchenne muscular dystrophy. Find out what it means for patients, payers, and global rollout.
Genethon advances Duchenne gene therapy trial while licensing Pompe tech to Bayer’s AskBio
Genethon advances gene therapies for Duchenne and Pompe disease. Find out what its nonprofit model and AskBio licensing deal mean for rare disease care.
Can ELEVIDYS meet its promise? Sarepta’s pivotal Duchenne gene therapy trial nears major milestone
Sarepta’s EMBARK 3-year readout could clarify ELEVIDYS’ long-term role in Duchenne care. Find out what’s at stake in this pivotal gene therapy trial.
FDA grants orphan drug designation to Atossa’s (Z)-Endoxifen for Duchenne: What’s next?
Atossa Therapeutics Inc. has received Orphan Drug Designation from the U.S. Food and Drug Administration for its investigational compound (Z)-Endoxifen in the treatment of Duchenne muscular dystrophy (DMD). The designation, disclosed on January 16, 2026, supplements the previously granted Rare Pediatric Disease Designation for the same compound and indication. The move marks a notable regulatory […]
What does Swissmedic approval of Santhera’s AGAMREE mean for long-term steroid treatment in Duchenne muscular dystrophy?
Swissmedic approves Santhera’s AGAMREE for Duchenne muscular dystrophy. Find out how vamorolone could reshape long-term steroid treatment strategies.