Because in #rarediseases, there is no money for small market cap companies (with no revenue) to pay for expanded access and in personalized biologics (unlike mass produced small molecule drugs unde CDER jurisdiction), it's even more difficult. Big Pharma donates drugs to their non-profit patient assistance progams and take tax writeoffs. But even Big Phama is reticent to use it on a broad scale. In #ALS, we passed a $500M bill called #ActforALS to fund research & EAPs yet it only helps a minute fraction of the 32k people with ALS. Thankfully the EAP data can help drug approvals -- but only if FDA agrees to consider the open label data. That's why BODs are loathe to use EAPs on large scale basis. Please don't assume they are "questionable drugs" when you don't know the data, the flawed clinical outcome assessments, the heteroegeneity of the disease, or the horrific burden of lethal diseases like ALS. Often, it's not the drug that doesn't work, but the flawed measurement tools, lack of biomarkers and short trial designs necessary because it's inhumane to keep dying patients on placebos for years. @houmanhemmati is right: the regulatory system is broken for terminal rare diseases.

The Clene Team joined @iamalsorg on the National Mall, where a sea of blue flags honors lives lost to ALS. We stand with families and support reauthorization of ACT for ALS to advance awareness, research, care, and a cure. 💙 #EndALS #ACTforALS

Thank you so much @JayObernolte for your cosponsorship of Act for ALS! Thank you Leg. Assistant Ashley for your role in getting us to this wonderful news today! 🙏🙏🙏 #ALS #ActForALS

The Act for ALS provided critical funding for research and expanded access to care. That legislation is set to expire in 2026. We stand with Eric Dane and the ALS community in the Push for Progress to ensure this funding is renewed. #ActForALS #ALSAwareness #ALSResearch

20 years ago today. 💔😢 #EricDane #ALS #ActForALS @TargetALS_fdn

His fight to raise awareness for ALS and funding for ALS research inspired so many. Eric Dane's voice will carry on. US Congress - Reauthorize ACT for ALS before it expires in September this year! #EricDane #ActForALS #ALS

The continued support from NINDS and the ACT for ALS initiative has been instrumental in enabling broader access to MN‑166 while capturing valuable clinical and biomarker insights. #MediciNova #ALSResearch #MN166 #ClinicalTrials #ExpandedAccess #ACTforALS

No words are adequate to describe the heartbreak of losing another #ALS warrior and friend Robert Survick. As an ASU gymnast and then coach with ASU and US Gymnastics, Rob touched so many young lives. He was kind, funny and a courageous warrior. When ALS slowly paralyzed his body, he continued to coach. He exemplified all the traits you'd want your sons to emulate and taught so many of us what it meant to live with courage, tenacity and purpose. For those of you new to the fight, Rob played a critical role in passing the #ACTforALS. So if you are getting drugs thru an Expanded Access Program, Rob and his wife Missy are part of the reason. They coordinated the US gymnastics community and all his student athletes around the country to contact Congress and pass the legislation to help 1000s of people. Rob was a huge sports fan -- especially the Arizona @Dbacks, and he was part of the team that successfully launched the first #LouGehrig Day here in Arizona. Rob also helped launch the first #DrugsInBodies campaign. To that end, he fought tirelessly for NurOwn's approval as he watched it help a fellow US gymnastics coach halt his progression. It's the ultimate tragedy that Rob represented our country around the world but our country blocked him from accessing the therapy that could help him live. @DrMakaryFDA @SecKennedy you can change this for the next person diagnosed with ALS. But more than a coach and an advocate extraordinaire, Rob was an amazing Dad and his family meant everything to him. Please keep his young kids and his wife Melissa Survick in your prayers. Fly high my friend. We will never forget you. @DHallDbacks @iamalsorg #endALS

⏱️ Countdown Family 🚨 I AM ALS is thenon-profit organization Eric Dane partnered with to launch the "Push for Progress" campaign for ALS research and to expand access to treatments. ACT for ALS is the law they're trying to renew. #EricDane #PushForProgress #ACTforALS

Countdown Family SAC, @RealEricDane, talking with CA Rep. Eric Salwell about the Push for Progress plan, getting funding for research, expand access to treatments and getting ACT for ALS reauthorized. @iamalsorg #ALS #ActForALS instagram.com/reel/DPjDN4ciP…

@RealEricDane’s honesty and strength in sharing his ALS journey move us all. His @washingtonpost interview shows the fight, the hope, and why we must keep going. Read the full interview below. #ActForALS #EndALS img.washingtonpost.com/style…

@RealEricDane's honesty and strength in sharing his ALS journey move us all. His @washingtonpost interview shows the fight, the hope, and why we must keep going. #ActForALS #EndALS img.washingtonpost.com/style…

@RealEricDane’s honesty and strength in sharing his ALS journey move us all. His @washingtonpost interview shows the fight, the hope, and why we must keep going. #ActForALS #EndALS img.washingtonpost.com/style…

@RealEricDane honesty and strength in sharing his ALS journey move us all. His @washingtonpost interview shows the fight, the hope, and why we must keep going. Read the full interview below. #ActForALS #EndALS img.washingtonpost.com/style…

Thank you @AndyOgles for taking our call. #ALS patients from #Tennessee appreciate your consideration for joining the Act For ALS. #FridayMorning @iamalsorg #ActForALS @VUMChealth #FridayVibes @MarshaBlackburn 🙏

Happy to have another opportunity to speak with @RepMikeQuigley about the reauthorization of the #ACTforALS. Stay tuned for more to come on this important piece of #ALS legislation.

7/ In closing, here's a project for #ALS journalists: file a FOIA request with @NIH, @NINDSfunding, @NINDStrials and ask which #ACTFORALS grants requests have been REJECTED since 2021. Let us know if you get a response and what it says. Deal? @statnews @biospace @alsnewstoday

1/ 🚨ALS Alert: Thanks to a wide eyed #NME member we can show you @MediciNova_US is 9.9% owned by the US division of the company that brought #ALS patients the #ALS "wunderdrug" #Radicava. Now, sadly an #ACTFORALS grant will fund an Ibudilast #EAP. Were #ALS patients asked? (Ctd)

Today I'm heartbroken to report that the #ALS community has lost a #USMC warrior, a leader in the #Veterans community and a great ALS advocate, Chris Mulholland, who died on January 24th. Chris was everything that was right about our country both because of his service and what he did after he retired from the USMC. When he was diagnosed, Chris was determined to leave the ALS & veterans community better than he found it. Chris said: “The person I was, I still am. So I will continue this fight until my dying breath." And that is what he did. As the National Deputy Chief of Staff for the @VFWHQ, he used that position to raise awareness about the increased risk to military men & women. He worked with the Veterans team at @iamalsorg to help Vets navigate the morass of paperwork to enable Veterans to get service-related benefits. In 2021, he authored an OpEd in the Virginian entitled "Veterans with ALS need Congress in the Fight." The next year he spread awareness further with an OpEd published in the Military Times entitled "ALS is Killing Veterans." He told the world: "According to a VA report, 1,055 veterans die from ALS each year. That means that since 9/11, three times more veterans have been lost to ALS than troops killed in combat in Iraq and Afghanistan combined (20,895 vs 6,896). Interestingly, the Hampton Roads area of southeastern Virginia — where I and 234,476 of my fellow veterans live — is a “hotspot” for ALS cases." (OpEds in comments). Personally, Chris and I couldn't have been more different politically. But rather than attacking each other, he understood that we could accomplish more together, so that is what we did. First he educated this civilian girl about the military. Then he joined me on dozens of zoom calls with Congress advocating for the #ActforALS, the Burn Pit bill & the Promising Pathway Act. Recently he continued his advocacy for the passage of the Elizabeth Dole Homecare Act. When we were trying to get the @US_FDA to give us an Advisory Committee meeting for #NurOwn, Chris helped me organize fellow veterans & then co-authored a Press Release and Open Letter to the FDA. In it he said: "Veterans with ALS have a unique stake in the fight for a NurOwn AdCom. First, we sacrificed our lives for every citizen's right of due process. It is the antithesis of all we fought for if we, now, were denied that same right..... Living daily with the inhumanity of this disease, General Mikolajcik challenged Congress and our country to step up its commitment to Veterans: 'If these soldiers were dying in the field rather than quietly at home… we would leave no stone unturned. We would use the best existing resources to make sure they had whatever they needed to survive… to ensure that no man or woman is left behind.... Please do not abandon us on this ALS battlefield.' " Thank you to Chris for all your sacrifices for your fellow Americans. We are sorry that our country abandoned you. Semper Fi my friend! USMC (Ret.) 1977-2000. Chris was deployed for combat tours in Iraq and Beirut. He deployed to Kuwait, Egypt, Haiti, Panama, Turkey, Morocco, and Japan. He was also stationed at Camp Lejeune. He was diagnosed with ALS in 2020. Please keep his family in prayer. @HouseVetAffairs @VetAffairsDems @SVACGOP @SVACDems @JerryMoran @timkaine @MarkWarner @SenRubenGallego @SenMarkKelly @SenDanSullivan @SenToddYoung @RepCarbajal @JeffersonShreve @sethmoulton @RepJackBergman @RepMaxMiller @RepTonyWied @RepAuchincloss @RepMikeBost @votevets @MilitaryTimes @VFW_OfficeDC

The #ALS community fought hard to pass the #ActforALS to fund EAPs. @MGHNeurology & the incomparable @sandymorris333 worked tirelessly to get manufacturers to understand the benefits of EAP. Join this HEALEY webinar on Thursday at 5 pm ET to see the fruits of all this hard work and to learn about the current EAPs. #EndALS