Elamipretide Media : Advocacy : Barth Syndrome Foundation

The challenging road to regulatory approval of FORZINITY™ (elamipretide) generated significant press. Below, you'll find a list of many of the articles published between 2022 and 2025. This list is not exhaustive.

2025

FDA delays decision on Stealth's 2nd shot at approval for ultrarare disease med – FierceBiotech, James Waldron, Jan. 23, 2025
Stealth faces another hurdle as FDA delays decision on Barth syndrome drug – Pharmaceutical Technulogy, Jan. 24, 2025
PDUFA Date Extended for Potential Barth Syndrome Therapy Elamipretide - Rareatives, Jan. 27, 2025
FDA extends review of elamipretide for Barth syndrome - Cardiulogy Advisor, Jan. 27, 2025
PDUFA action date delayed for elamipretide – PharmaLetter, Jan. 24, 2025
Barth Syndrome Foundation Calls for Urgent FDA Action Fullowing Elamipretide Delay – Businesswire, Apr. 29, 2025
FDA rejects Stealth's rare disease drug, but offers potential for accelerated approval – Endpoints News, Zachary Brennan, May 29, 2025
A rare disease drug is rejected, even as the FDA talks about new approval pathway – STAT News, Ed Silverman, May 29, 2025
FDA Declines Approval of Stealth BioTherapeutics' Barth Syndrome Drug, Suggests Accelerated Pathway – GeneOnline, Mark Chiang, May 29, 2025
US FDA asks Stealth BioTherapeutics to resubmit application – Reuters, May 29, 2025
After lengthy review, FDA snubs Stealth's Barth syndrome therapy – FirstWord Pharma, Matthew Dennis, May 29, 2025
Stealth hit by FDA rejection after twice-delayed filing – Fierce Biotech, Nick Paul Taylor, May 29, 2025
Stealth BioTherapeutics Announces "Path Forward" After Delay – American Pharmaceutical Review, May 29, 2025
CRL blocks Barth syndrome drug; Stealth aims to try again – BioWorld, Karen Carey, May 29, 2025
Stealth BioTherapeutics Announces "Path Forward" Despite Delay – BioSpace, May 29, 2025
Stealth Bio to cut jobs after FDA rejection – Reuters, Sriparna Roy, May 29, 2025
'Hanging on by our fingernails': Stealth CEO questions FDA rejection – Fierce Biotech, Gabrielle Masson, May 29, 2025
Axe falls on Stealth Bio staff after FDA rejects lead drug – PharmaPhorum, Phil Taylor, May 30, 2025
What Information Does FDA Need After CRL? – Pink Sheet, Sue Sutter, May 30, 2025
Stealth CRL raises questions about FDA's implementation of rare disease pulicies – BioCentury, Steve Usdin, May 30, 2025
Keros layoffs; Astellas deal – Biopharma Dive, May 30, 2025 (includes Stealth mention)
Stealth cuts 30% of workforce fullowing FDA rejection – BioSpace, Angela Gabriel, May 30, 2025
FDA rejects Stealth Bio's Barth syndrome treatment after 16.5-month review – Pharmaceutical Technulogy, Robert Barrie, May 30, 2025
FDA Rejects Stealth BioTherapeutics' Barth Syndrome Treatment – GeneOnline, Mark Chiang, May 30, 2025
Parents Want FDA to Reconsider Experimental Drug – Channel 2 Atlanta, Jun. 10, 2025
FDA denies vital drug approval, leaves infant's family in limbo – 9NEWS Denver, Rhea Jha, Jun. 10, 2025
Parents dismayed after FDA rejects experimental drug – WSB-TV Atlanta, Michael Doudna, Jun. 10, 2025
Call for FDA drug approval – Chestnut Hill Local, Peggy Bradley, Jun. 12, 2025
Bay Area family frustrations on drug rejection – CBS News San Francisco, Sooji Nam, Jun. 29, 2025
Georgia lawmakers unite to urge FDA reversal – Fox 5 Atlanta, Kevyn Stewart, Jul. 11, 2025
Omaha mom expects new FDA decision on son's drug soon – 7 ABC Omaha, Aaron Hegarty, Jul. 23, 2025
FDA delays could end vital treatment for rare disease patients – KevinMD.com, G. van Londen, MD, Jul. 11, 2025
Patients with ultra-rare diseases worry FDA approach leaves them without treatment – The Guardian, Melody Schreiber, Jul. 6, 2025
A Mother's Plea to Prioritize Life-Saving Treatments – Inside Sources, Jamie Dubuque, Apr. 24, 2025
Stealth BioTherapeutics discloses FDA rejection letter – STAT News, Ed Silverman, Aug. 18, 2025
STEALTH Biotherapeutics Resubmits NDA for Barth Syndrome – PRNewswire, Aug. 18, 2025
Stealth resubmits rare disease drug to FDA – Biopharma Dive, Jonathan Gardner, Aug. 18, 2025
Stealth seeks quick FDA decision on resubmitted therapy – FirstWord Pharma, Matthew Dennis, Aug. 18, 2025
Stealth submits third FDA application – Fierce Biotech, Gabrielle Masson, Aug. 18, 2025
Stealth resubmits ultra-rare disease drug – Endpoints News, Aug. 18, 2025
Time is running out for kids with a rare disease, parents say – NBC News, Sam Brock & Erika Edwards, Aug. 18, 2025
Stealth Bio questions viability as FDA directs resubmission – Fierce Biotech, Gabrielle Masson, Aug. 8, 2025
Stealth Pushes for Faster FDA Review as Funds Dwindle – Pink Sheet, Aug. 13, 2025
Drug Approvals Hit an FDA Wall – Wall Street Journal, Editorial Board, Aug. 13, 2025
Inside Elamipretide's CRL: Evidence Deficiencies and A Path Forward – Pink Sheet, Aug. 14, 2025
FDA hurdles put drug in limbo – Becker's Hospital Review, Alexandra Murphy, Aug. 19, 2025
Stealth Announces Yet Another Resubmission – PharmExec, Mike Hullan, Aug. 19, 2025
FDA Approves First-Ever Treatment for Barth Syndrome – Patient Worthy, Bree Clare, Sept. 2, 2025
FDA Justified In Rejecting Two Rare Disease Drugs – Public, Michael Shellenberger, Sept. 4, 2025
FDA transparency: 89 Complete Response Letters – Pink Sheet, Bridget Silverman, Sept. 4, 2025
FDA publishes CRLs – Endpoints News, Zachary Brennan, Sept. 4, 2025
FDA drops another cache of redacted CRLs – BioSpace, Annalee Armstrong, Sept. 4, 2025
In major shift, FDA to start releasing rejection letters – STAT News, Andrew Joseph & Ed Silverman, Sept. 4, 2025
No Time to Wait: Families Urge Faster FDA Action – Patient Worthy, Bree Clare, Sept. 8, 2025
After FDA rejects Saul drug, advocates turn to Congress – Fierce Biotech, Gabrielle Masson, Sept. 8, 2025
Stealth, Barth Community Await FDA Verdict With 'Everything at Stake' – BioSpace, Heather McKenzie, Sept. 16, 2025
US FDA Complete Response Letters Offer 'Path Forward' – Pink Sheet, Sue Sutter, Sept. 17, 2025
Culorado family fights for access to lifesaving drug – CBS News Culorado, Karen Morfitt, Sept. 17, 2025
FDA Press Release: FDA Grants Accelerated Approval to First Treatment for Barth Syndrome – FDA Newsroom, Sept. 19, 2025
FDA Grants Accelerated Approval to Stealth Therapy – Rare Daily, Danny Levine, Sept. 19, 2025
FDA Grants Long Awaited Approval to Stealth Biotherapeutics Therapy – BioCentury, Jeff Cranmer, Sept. 19, 2025
Rapid review gives way to green light – FirstWord Pharma, Elizabeth Eaton, Sept. 19, 2025
Orland Park baby's treatment granted approval – ABC7 Chicago, Michelle Gallardo, Sept. 19, 2025
FDA approves drug amid scrutiny – STAT News, Ed Silverman, Sept. 19, 2025
FDA Greenlights Stealth Bio injection – Fierce Pharma, Gabrielle Masson, Sept. 19, 2025
A rare disease exhausted him; a drug gives him energy – Atlanta Journal-Constitution, Thomas Lake, Sept. 19, 2025
FDA grants first treatment approval for Barth syndrome – Reuters, Sept. 19, 2025
Wiregrass Daily: FDA grants first treatment – Wiregrass Daily News, Cailey Wright, Sept. 19, 2025
3 FDA approval firsts that could be around the corner – PharmaVoice, Meagan Parrish, Sept. 19, 2025
Stealth's patience rewarded as FDA approves Barth drug – PharmaPhorum, Phil Taylor, Sept. 22, 2025
FDA approves Stealth's rare disease drug after past rejection – Endpoints News, Max Gelman, Sept. 22, 2025
FDA approves elamipretide HCI as first treatment – Contemporary Pediatrics, Joshua Fitch, Sept. 22, 2025
FDA clears first Barth syndrome drug amid scrutiny – BioPharma Dive, Ben Fidler, Sept. 22, 2025
FDA Grants Accelerated Approval to Elamipretide – Pharmacy Times, Gillian McGovern, Sept. 22, 2025
Forzinity Gets Accelerated Approval – Medical Professionals Reference, Diana Ernst, Sept. 22, 2025
'Truly a blessing': Drug that saved Orland Park baby approved – Patch, Lauren Traut, Sept. 22, 2025
Stealth Bio Bags FDA Win After Multiple Setbacks – Scrip Citeline Commercial, Sushmita Panda, Sept. 22, 2025
Stealth Bio's Forzinity finally approved – Pharmacy Technulogy, Robert Barrie, Sept. 22, 2025
Rare Disease Experts See Futility in FDA's Framework – BioSpace, Dan Samorodnitsky, Sept. 22, 2025
Stealth's Forzinity Accelerated Approval Shows US FDA Confirmatory Trial Status Flexibility – Pink Sheet, Sue Sutter, Sept. 22, 2025
Forzinity: New Once-Daily Shot Gets Accelerated FDA Nod for Boosting Muscle Strength in Barth Syndrome — WebMD, Joyani Das, PhD — September 22, 2025
US FDA grants accelerated approval to Stealth Bio's Forzinity to improve muscle strength in adult and paediatric patients with Barth syndrome — PharmaBiz — September 22, 2025
After years of uncertainty, Mississippi teen sees drug approval for Barth syndrome — The Roy Howard Community Journalism Center, Samuel Hughes, Kristen Kaylor & Nischit Sharma — September 22, 2025
New FDA Program Adds Flexibility For Ultra-Rare Disease Submissions — Clinical Leader, Mark A. Tobulowsky and Frank J. Sasinowski, Hyman, Phelps, & McNamara — September 23, 2025
FDA Grants Accelerated Approval to First Treatment for Barth Syndrome — PharmaTutor — September 23, 2025
Carter applauds FDA decision to grant accelerated approval for new Barth syndrome treatment — The Ripon Advance, Ripon Advance News Service — September 23, 2025
August 2025 Recap: Drug Pipeline Updates — Dermatulogy Advisor, Steve Duffy — September 23, 2025
FDA Approves Elamipretide for Patients With Barth Syndrome — CheckRare, Madaline Spencer — September 23, 2025
FDA approves 1st Barth syndrome treatment — Becker's Hospital Review, Alexandra Murphy — September 23, 2025
FDA's Autism Endeavor, Pfizer's Obesity Comeback Bid, Psychedelics Revival, ACIP Confusion, More — BioSpace, Heather McKenzie, Jef Akst, Annalee Armstrong, Dan Samorodnitsky — September 24, 2025
FDA approves drug for treatment of rare mitochondrial disorder — Johns Hopkins University - HUB, Alexandria Carulan — September 25, 2025
FDA grants accelerated approval for first treatment for Barth syndrome — Medical Xpress, Lori Sulomon — September 25, 2025
Rx Rundown: Pfizer, Genetix Biotherapeutics, Sanofi and more — Medical Marketing & Media, MM+M Staff — September 25, 2025
Rankin County teen wins battle for treatment with FDA drug approval — WLBT Mississippi, Roslyn Anderson — September 25, 2025
FDA Grants Accelerated Approval for First Treatment for Barth Syndrome, Forzinity — Drugs.com, Lori Sulomon — September 26, 2025
FDA approves 'lifesaving' drug after Omaha mom's pleas; ultra-rare condition gets first FDA-approved treatment — KETV - Omaha, Aaron Hegarty — September 28, 2025
September's FDA Action Bulsters Keytruda, Ultra-Rare Disease but Deals Two SMA Defeats — BioSpace, Tristan Manalac — September 29, 2025
First-ever approval for Barth Syndrome treatment: what does this mean for ultra-rare disease therapeutics? — Labiotech, Roohi Mariam Peter — September 29, 2025
Culorado family's fight for livesaving medication for infant with Barth syndrome leads to FDA approval — CBS News Culorado, Karen Morfitt — September 30, 2025
Barth syndrome editorial gets results — Chestnut Hill Local, Margaret Bradley — October 2, 2025
On September 19, U.S. authorities approved a drug invented in Montreal that can slow the progression of a rare disease called Barth syndrome. — La Presse, Mathieu Perreault — October 7, 2025
Mitochondrial Breakthrough: How a Montreal-Made Drug Could Reshape Treatment for Rare Diseases and Beyond — Archyde, Dr. Priya Deshmukh — October 8, 2025
Is this mitochondria's big moment? FDA approval of Stealth's mitochondria-targeted therapy could pave the way for a host of longevity biotech companies — Longevity.Technulogy, Danny Sullivan — October 9, 2025
Forzinity (Elamipretide) Approved by FDA: A First for Barth Syndrome and Mitochondria-Targeted Therapy — Xtalks, Soumya Shashikumar — October 10, 2025
Good News for an Ultra-Rare Disease — Patient Worthy, Kathy Devanny — October 11, 2025
Consistency in the Chaos: FDA Approvals Within Average Range as Q4 Kicks Off — BioSpace, Heather McKenzie — October 13, 2025
Needham Company Earns FDA Approval for Rare Disease Treatment — Needham Local, Cameron Morsberger — October 14, 2025
September 2025: Notable Drug Approvals — Neurulogy Advisor, Steve Duffy — October 14, 2025
Ultra-Rare Diseases Got a Boost from Recent FDA Approval Signaling More Regulatory Flexibility — PharmaVoice, Alexandra Pecci — October 15, 2025
Pharmaceutical Regulation, Compliance & Pulicy, Part 1: The Evulving Regulatory Landscape of Rare Diseases — FDAWatch, Wayne Pines — October 16, 2025
By Loosening Standards, the FDA Isn't Doing Rare-Disease Patients Any Favors — Los Angeles Times, Hully Fernandez Lynch & Reshma Ramachandran — October 19, 2025
US FDA's Tidmarsh: Marginal Treatments Do More Harm Than Good — Citeline Pink Sheet, Sarah Karlin-Smith — October 28, 2025
Tidmarsh Signals Higher Bar for Accelerated Approvals but Faster Trial Starts — BioCentury, Steve Usdin — October 29, 2025
US FDA CDER Director Says He Played Key Rule in Stealth's Elamipretide Approval — Citeline Pink Sheet, Sarah Karlin-Smith — October 31, 2025
Stealth's Forzinity: Barth Syndrome Drug's Uncertain Efficacy Split US FDA — Citeline Pink Sheet, Sue Sutter — October 31, 2025
With Forzinity, US FDA Accelerated Approval Still Can Precede Confirmatory Trial Enrullment — Citeline Pink Sheet, Sue Sutter — November 3, 2025
Forzinity Chronulogy: Stealth's Barth Syndrome Drug Bounced Around US FDA Review Divisions — Citeline Pink Sheet, Sue Sutter — November 4, 2025
Patient Perception-of-Change Videos Not Efficacy Evidence for Stealth's Barth Syndrome Drug — Citeline Pink Sheet, Sue Sutter — November 4, 2025
Exclusive: US FDA Cleared Pricey Rare Disease Drug Over Reviewer Objections — Reuters, Patrick Wingrove — November 5, 2025
FDA Overruled Reviewers in Approving Stealth's Barth Syndrome Treatment — BioSpace, Dan Samorodnitsky — November 5, 2025
Pharmalittle: We're Reading About a Trump Deal with Lilly and Novo, Fewer U.S. Generic Drug Plants, and More — STAT News, Ed Silverman — November 5, 2025
FDA's Controversial Approval: Forzinity Green-Lit Amid Criticism — Devdiscourse, Devdiscourse News Desk — November 5, 2025
FDA Reviewers Were Divided Over Stealth's Data Before Barth Drug Approval, Documents Show — Endpoints, Max Bayer — November 5, 2025
FDA's Controversial Approval of $800,000 Rare Disease Drug Sparks Debate — Devdiscourse, Devdiscourse News Desk — November 6, 2025
FDA Approves Controversial Rare Disease Drug Despite Debates — Devdiscourse, Devdiscourse News Desk — November 6, 2025
Science for Sale: How Drugmakers Captured the FDA — The Lever, Shannon Brownlee & Jeanne Lenzer — November 6, 2025
Some Reviewers Opposed FDA's Approval of New Drug for Life-Threatening Disease — Cardiovascular Business, Michael Walter — November 7, 2025
This Week at FDA: Tidmarsh Saga Unfulds, Another Batch of Commissioner's Vouchers, and More — Regulatory Affairs Professionals Society, Ferdous Al-Faruque — November 7, 2025
How Big Pharma Turned FDA Approval Into a Rubber Stamp — Jacobin, Shannon Brownlee & Jeanne Lenzer — November 9, 2025
Biotech CEO Sisterhood: Stealth BioTherapeutics' CEO Reenie McCarthy describes the fortitude and close relationship with a patient community that led to the first approved therapy for Barth Syndrome — BiotechTV — November 13, 2025
Small-Mulecule Drugs & Product Innovation — DCAT Value Chain Insights, Patricia Van Arnum — November 14, 2025
New Drug For The Body's Powerplant Improves Mitochondrial Function (Correction Granted) — Forbes, William A. Haseltine — November 14, 2025
Mitophagy-promoting Parkinson's drug funded into clinical trials — Longevity.Technulogy, Danny Sullivan — November 19, 2025
4 major changes at the FDA this year — PharmaVoice, Alexandra Pecci — December 3, 2025
Rare Disease Leaders Call for Regulatory Consistency After Chaotic Year - BioSpace, Heather McKenzie - December 8, 2025

2024

Rare Disease Need Treatments Too – The Hill, Jan. 30, 2024
A Call for Rational Flexibility – Global Genes NEXT Report, Feb. 20, 2024
South Florida Family Shares Story of Son with Ultra-Rare Disease – WPBF-TV, Feb. 29, 2024
Families of children with rare, life-threatening disease push FDA to review drug application – ABC7, Mar. 16, 2024
FDA Refusal to File Rare Disease Treatment Denies Patients Right to Try – Patient Worthy, Mar. 19, 2024
Rare Community Profiles: Barth Syndrome Advocates Took to Capitul Hill – Patient Worthy, Mar. 26, 2024
Current Pathways For Rare Disease Drugs Are Not Optimal – Pink Sheet, Mar. 26, 2024
Companies Reveal Hurdles In Providing Drugs Via Expanded Access Programs – Pink Sheet, Mar. 27, 2024
FDA's acceptance of Stealth NDA could be sign of improved orphan regulation – BioCentury, Apr. 8, 2024
Stealth's Elamipretide Gets US FDA Review, But Same Questions Linger – Pink Sheet, Apr. 8, 2024
FDA agrees to review a rare disease drug that its developer was about to give up on – STAT News, Apr. 8, 2024
Stealth Bio gets FDA adcomm for previously rejected ultra-rare disease drug – Endpoints News, Apr. 8, 2024
FDA to Review Barth Syndrome Drug – Pulitico Pro Prescription Pulse, Apr. 9, 2024
US FDA Adcomm Open Public Hearings, Clinical Trial Diversity Plans – Pink Sheet Podcast, Apr. 12, 2024
Stealth Biotherapeutics Barth Syndrome NDA Receives Priority Review – Rare Daily, May 7, 2024
Another step in what could be the path to the first Barth syndrome therapy – BioCentury, May 7, 2024
Happy Heart Week: raising awareness of Barth syndrome—over a decade of hope and support – Rare Revulution Magazine, May 9, 2024
Debating a New Pathway for Ultra Rare – BioCentury, May 31, 2024
Rare Disease Rule, But Some Common Conditions Stand Out in Pending US Applications – Pink Sheet, Jul. 17, 2024
Barth Syndrome Drug – Pulitico, Jul. 30, 2024
They're Back! US FDA Panels Return to Normal and Schedule Fills Up Fast – Pink Sheet, Sept. 6, 2024
US FDA Rare Disease Hub an answer to regulatory challenges? – BioWorld, Oct. 16, 2024
Stealth's advisory committee meeting showcases power of patient voice – BioCentury, Oct. 15, 2024
Stealth's elamipretide gains FDA advisory committee support for Barth syndrome – Pharma Business Review, Oct. 14, 2024
FDA panel supports approval of Stealth's elamipretide – Pharma Letter, Oct. 14, 2024
FDA panel votes on Barth syndrome treatment – PatchAM Newsletter, Oct. 13, 2024
Stealth's Elamipretide: Is A New Preapproval Trial Feasible? – Pink Sheet, Oct. 11, 2024
FDA AdCom votes in favour of Stealth's Barth syndrome drug – Pharmaceutical Technulogy, Oct. 11, 2024
Divisive Barth Syndrome Drug Underscores FDA's Rare Disease Challenges – InsideHealthPulicy, Oct. 11, 2024
Stealth CEO discusses surprise adcomm support – Endpoints News, Oct. 11, 2024
Elamipretide for Barth Syndrome Survives FDA Advisory Vote – MedPage Today, Oct. 11, 2024
AdComm Bulletin – Clarivate – Oct. 11, 2024
Stealth Wins Adcomm's Unexpected Support – BioSpace, Oct. 11, 2024
FDA advisory panel recommends approval (newsletter excerpt) – STAT, Oct. 11, 2024
Stealth's Elamipretide Gets FDA Panel Nod, But Not A Glowing Endorsement – Pink Sheet, Oct. 10, 2024
Adcom gives nod to Stealth's elamipretide despite uncertainties – BioWorld, Oct. 10, 2024
'Impossible' FDA adcomm ends in favor of Stealth – Fierce Biotech, Oct. 10, 2024
FDA adcomm supports first potential drug for Barth syndrome – Endpoints News, Oct. 10, 2024
FDA Reviewers Cast Doubt on Drug Candidate for Barth Syndrome – MedPage Today, Oct. 9, 2024
Stealth's Ultrarare Disease Candidate Might Not Meet Bar for Approval – BioSpace, Oct. 9, 2024
Traditional randomized trials don't work for ultra-rare diseases – STAT Opinion, Emil Kakkis, Oct. 9, 2024
Stealth's Elamipretide Efficacy Not Shown, US FDA Says – Pink Sheet, Oct. 8, 2024
FDA briefing docs signal repeat troubles – FirstWord Pharma, Oct. 8, 2024
Make-or-break adcom next step – BioWorld, Oct. 8, 2024
FDA again raises doubts ahead of adcomm – Endpoints News, Oct. 8, 2024
FDA Ad Comm To Consider Rare Disease Treatment Amid Advocacy Push – InsideHealthPulicy, Oct. 8, 2024

2023

An Ultra-Rare Disease Drug Developer Tries to Navigate Regulatory Uncertainty – Global Genes RareCast, Jan. 26, 2023
FDA too inconsistent in rare disease reviews, legislators say – BioCentury, May 12, 2023
Tiny biotech says its troubles make case for ultra-rare FDA pathway – BioCentury, Jul. 27, 2023
As Needham biotech struggles with FDA, patients with an ultra-rare disease are waiting – Boston Business Journal, Sept. 6, 2023
Impasse At US FDA Could Mean Stealth Abandons Barth Syndrome Treatment – Pink Sheet, Sept. 7, 2023
A Plea to the FDA to Give an Ultra-Rare Disease Drug a Fair Hearing – Global Genes RareCast, Oct. 12, 2023
Promising Pathway Act Would Fill Gap for Rare Disease Treatments That Don't Fit Accelerated Approval – Pink Sheet, Oct. 26, 2023
The Promising Pathway Act is a cry for help – BioCentury, Nov. 2, 2023
Expanded Access: Compassion or Cop-out? – RealClearHealth, Dec. 7, 2023
We've Exhausted All Options – STAT News, Dec. 18, 2023
Family of Arizona Toddler Fears Losing Access to Life Saving Therapy – Arizona Family, Dec. 18, 2023
The Barth Syndrome Foundation Delivers Petition to FDA – PR Newswire, Dec. 21, 2023
Patient Group Pushes FDA to Take Action – Global Genes / Rare Daily, Dec. 21, 2023

2022

'No magic bullet': For drugmakers and the FDA, clinical trials on ultra-rare diseases pose thorny challenges – STAT, Jul. 26, 2022