Really great article about the diversity in the MASH space - not enough attention to antioxidant therapies though... #pediatric #MASH #oxidativestress #mitochondrialdysfunction pharmavoice.com/news/mash-mark…

PTC Therapeutics $PTCT agreed to sell its Rare Pediatric Disease Priority Review Voucher for $150 million. The PRV was granted on November 13, 2024, with FDA approval of KEBILIDI for AADC deficiency. ir.ptcbio.com/news-releases/…

$LLY Lilly's Zepbound superior to Wegovy (semaglutide) in head-to-head trial showing an average weight loss of 20.2% vs. 13.7%. $NVO Participants using Zepbound lost 50.3 lbs (22.8 kg) and participants on Wegovy lost 33.1 lbs (15.0 kg) prnewswire.com/news-releases/…

Great to see the strong interest in new compounds that enhance GLP1 drugs. $TTI.v $MASH $GSH fiercebiotech.com/biotech/novo-h…

Historical investor return is 2.6x with a 15% annual rate of return for acquired companies (2005-2020 data set) The ideal profile is an orphan, multi-indication, small-molecule, oncology asset #learnbiotechinvesting #biotech #investing #BiotechPrometheus

Mitochondrial trials update. $TTI.v #UMDF #mitochondria #Leigh #MELAS newsfilecorp.com/release/233143…

The Priority Review Voucher Program #PRV is set to expire in ONE WEEK. Families across the nation are urging you to take action to support the #PRVProgram, ensuring the continued development of life-saving treatments! Time is critical—act now to help secure #Cures4RareKids:

The United Mitochondrial Disease Foundation (UMDF) Board of Trustees has named Kristen Clifford the organization’s next President and Chief Executive Officer, effective January 13. Read more here: umdf.org/umdf-names-new…

We are thrilled to welcome Thiogenesis Therapeutics Corp. (TSX-V: $TTI; OTCQX: $TTIPF), a clinical-stage biotech company, to our #OTCQX Best Market. Congratulations to the Thiogenesis Therapeutics team! Visit the company's profile to learn more: bit.ly/41bm5Sc

Congratulations to $SLNO - always great to see an orphan approval for an unmet need.

Today is #ClinicalTrialsDay, a moment to honor the researchers, coordinators, and especially the PARTICIPANTS who make progress possible. Thank you to all who advance hope toward treatments for our community with #mitochondrialdisease. Visit umdf.org/clinical-trials

newsfilecorp.com/release/251971… $TTI $TTIPF $MELAS $Mitochondria

With over 95% of rare diseases lacking approved therapies, the ORPHAN Cures Act would be a game-changer for the rare disease community. RDCC thanks Sen. John Barrasso and Martin Heinrich for their leadership, and we will continue our work to advance this important policy.

Health and Human Services Secretary Robert F. Kennedy Jr. said on Thursday that the U.S. drugs regulator would look for ways to fast-track approval for rare disease treatments and remove obstacles to their path to market. reuters.com/business/healt…

Great news today - excited to clear IND for LSS. $TTI.v $TTIPF #mitochondria #Leighsyndrome #cysteine #glutathione #IND newsfilecorp.com/release/255101…

Thiogenesis is excited to start dosing MELAS patients in France, looking forward to interim data in September. $TTI.v $TTIPF #mito #MELAS #Angers newsfilecorp.com/release/255687…

Looking forward to a good turnout at MitoMed 2025. $TTI.v $TTIPF #mito #Leigh's #melas newsfilecorp.com/release/256143…

Looking forward to filing IMPD for a Ph 2a pediatric MASH trial in the EU - an unserved market for kids with severe liver disease. $TTIV $TTIPF #mito #fattyliver #oxidativestress newsfilecorp.com/release/256736…

The ORPHAN Cures Act will enable more options for Americans living with rare disease. Congress must pass the #ORPHANCuresAct to ensure continued investment in lifesaving treatments and to protect U.S. #biotech leadership.

The FDA has accepted Stealth BioTherapeutics’ third new drug application for its ultrarare disease candidate, providing a planned decision date of Sept. 26. fiercebiotech.com/biotech/fda-ac…